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Europe approves first gene therapy for hemophilia B
The approval means that the product will now be available in all the countries of the European Union as well as the European Economic Area.
The gene therapy was approved in the United States in November 2022. It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.
The treatment comprises a one-time infusion of a functional gene that acts as a blueprint for coagulation factor IX, a protein important for blood clotting, stated the manufacturer, CSL.
People living with hemophilia B currently require lifelong treatment of intravenous infusions of factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and well-being, the company explained in its press release.
The approval was based on findings from the pivotal HOPE-B trial, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; first results from this trial were reported at the 2020 annual meeting of the American Society of Hematology.
The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity (with a mean factor IX activity of 36.9%), which led to an adjusted annualized bleeding rate reduction of 64%.
After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post treatment compared with the lead-in period, the company noted.
“Data from the HOPE-B study demonstrate the potential of Hemgenix to remove the need for routine prophylaxis by providing durable factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B,” said one of the trialists, Wolfgang Miesbach, MD, PHD, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.
This European approval “marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints, and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,” Dr. Miesbach said in the company press release.
A version of this article first appeared on Medscape.com.
The approval means that the product will now be available in all the countries of the European Union as well as the European Economic Area.
The gene therapy was approved in the United States in November 2022. It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.
The treatment comprises a one-time infusion of a functional gene that acts as a blueprint for coagulation factor IX, a protein important for blood clotting, stated the manufacturer, CSL.
People living with hemophilia B currently require lifelong treatment of intravenous infusions of factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and well-being, the company explained in its press release.
The approval was based on findings from the pivotal HOPE-B trial, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; first results from this trial were reported at the 2020 annual meeting of the American Society of Hematology.
The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity (with a mean factor IX activity of 36.9%), which led to an adjusted annualized bleeding rate reduction of 64%.
After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post treatment compared with the lead-in period, the company noted.
“Data from the HOPE-B study demonstrate the potential of Hemgenix to remove the need for routine prophylaxis by providing durable factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B,” said one of the trialists, Wolfgang Miesbach, MD, PHD, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.
This European approval “marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints, and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,” Dr. Miesbach said in the company press release.
A version of this article first appeared on Medscape.com.
The approval means that the product will now be available in all the countries of the European Union as well as the European Economic Area.
The gene therapy was approved in the United States in November 2022. It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.
The treatment comprises a one-time infusion of a functional gene that acts as a blueprint for coagulation factor IX, a protein important for blood clotting, stated the manufacturer, CSL.
People living with hemophilia B currently require lifelong treatment of intravenous infusions of factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and well-being, the company explained in its press release.
The approval was based on findings from the pivotal HOPE-B trial, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; first results from this trial were reported at the 2020 annual meeting of the American Society of Hematology.
The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity (with a mean factor IX activity of 36.9%), which led to an adjusted annualized bleeding rate reduction of 64%.
After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post treatment compared with the lead-in period, the company noted.
“Data from the HOPE-B study demonstrate the potential of Hemgenix to remove the need for routine prophylaxis by providing durable factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B,” said one of the trialists, Wolfgang Miesbach, MD, PHD, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.
This European approval “marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints, and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,” Dr. Miesbach said in the company press release.
A version of this article first appeared on Medscape.com.
FDA approves once-weekly hemophilia A product
The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).
The manufacturer, Sanofi, says that this is “the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.”
With this product, “we have the opportunity to provide near-normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” said Angela Weyand, MD, of Michigan Medicine, who was involved in the pivotal phase 3 XTEND-1 trial that led to approval.
Results from the XTEND-1 trial were recently published online in The New England Journal of Medicine. The results show that one injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week, as previously reported by this news organization.
The researchers noted that the factor VIII therapies that have been available up to now need to be administered frequently.
In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.
“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” wrote Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University in New Orleans.
Sanofi said that Altuviiio is expected to be commercially available in April 2023. The company said that it will price the product at parity to the annual cost of treating a prophylaxis patient with its Eloctate product (antihemophilic factor [recombinant], Fc fusion protein). Sanofi will provide comprehensive patient support services and resources online and at 1-855-MyALTUVIIIO (855-692-5888).
A version of this article originally appeared on Medscape.com.
The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).
The manufacturer, Sanofi, says that this is “the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.”
With this product, “we have the opportunity to provide near-normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” said Angela Weyand, MD, of Michigan Medicine, who was involved in the pivotal phase 3 XTEND-1 trial that led to approval.
Results from the XTEND-1 trial were recently published online in The New England Journal of Medicine. The results show that one injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week, as previously reported by this news organization.
The researchers noted that the factor VIII therapies that have been available up to now need to be administered frequently.
In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.
“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” wrote Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University in New Orleans.
Sanofi said that Altuviiio is expected to be commercially available in April 2023. The company said that it will price the product at parity to the annual cost of treating a prophylaxis patient with its Eloctate product (antihemophilic factor [recombinant], Fc fusion protein). Sanofi will provide comprehensive patient support services and resources online and at 1-855-MyALTUVIIIO (855-692-5888).
A version of this article originally appeared on Medscape.com.
The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).
The manufacturer, Sanofi, says that this is “the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.”
With this product, “we have the opportunity to provide near-normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” said Angela Weyand, MD, of Michigan Medicine, who was involved in the pivotal phase 3 XTEND-1 trial that led to approval.
Results from the XTEND-1 trial were recently published online in The New England Journal of Medicine. The results show that one injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week, as previously reported by this news organization.
The researchers noted that the factor VIII therapies that have been available up to now need to be administered frequently.
In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.
“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” wrote Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University in New Orleans.
Sanofi said that Altuviiio is expected to be commercially available in April 2023. The company said that it will price the product at parity to the annual cost of treating a prophylaxis patient with its Eloctate product (antihemophilic factor [recombinant], Fc fusion protein). Sanofi will provide comprehensive patient support services and resources online and at 1-855-MyALTUVIIIO (855-692-5888).
A version of this article originally appeared on Medscape.com.
FDA broadens warning on potentially contaminated eye products
The announcement released Wednesday adds to a previous warning issued earlier this month for EzriCare Artificial Tears or Delsam Pharma’s Artificial Tears because of potential bacterial contamination. All three products are manufactured by the same company, Global Pharma Healthcare, based in Tamilnadu, India.
The FDA has faulted the company for multiple violations, including “lack of appropriate microbial testing” and “lack of proper controls concerning tamper-evident packaging,” and has banned imports to the United States.
The updated warning from the FDA did not give additional information about the over-the-counter eye ointment beyond potential bacterial contamination.
On Feb. 1, the CDC issued an alert about an outbreak of a drug-resistant strain of bacteria, Pseudomonas aeruginosa, linked to artificial tear products. To date, 58 patients across 13 states have been identified, and the most commonly reported artificial tear brand was EzriCare Artificial Tears. Five patients had permanent vision loss, and one patient died.
A version of this article first appeared on Medscape.com.
The announcement released Wednesday adds to a previous warning issued earlier this month for EzriCare Artificial Tears or Delsam Pharma’s Artificial Tears because of potential bacterial contamination. All three products are manufactured by the same company, Global Pharma Healthcare, based in Tamilnadu, India.
The FDA has faulted the company for multiple violations, including “lack of appropriate microbial testing” and “lack of proper controls concerning tamper-evident packaging,” and has banned imports to the United States.
The updated warning from the FDA did not give additional information about the over-the-counter eye ointment beyond potential bacterial contamination.
On Feb. 1, the CDC issued an alert about an outbreak of a drug-resistant strain of bacteria, Pseudomonas aeruginosa, linked to artificial tear products. To date, 58 patients across 13 states have been identified, and the most commonly reported artificial tear brand was EzriCare Artificial Tears. Five patients had permanent vision loss, and one patient died.
A version of this article first appeared on Medscape.com.
The announcement released Wednesday adds to a previous warning issued earlier this month for EzriCare Artificial Tears or Delsam Pharma’s Artificial Tears because of potential bacterial contamination. All three products are manufactured by the same company, Global Pharma Healthcare, based in Tamilnadu, India.
The FDA has faulted the company for multiple violations, including “lack of appropriate microbial testing” and “lack of proper controls concerning tamper-evident packaging,” and has banned imports to the United States.
The updated warning from the FDA did not give additional information about the over-the-counter eye ointment beyond potential bacterial contamination.
On Feb. 1, the CDC issued an alert about an outbreak of a drug-resistant strain of bacteria, Pseudomonas aeruginosa, linked to artificial tear products. To date, 58 patients across 13 states have been identified, and the most commonly reported artificial tear brand was EzriCare Artificial Tears. Five patients had permanent vision loss, and one patient died.
A version of this article first appeared on Medscape.com.
Zika virus still calls for preparedness and vaccine development
Warming U.S. temperatures, the resumption of travel, and new knowledge about Zika’s long-term effects on children signal that Zika prevention and vaccine development should be on public health officials’, doctors’, and communities’ radar, even when community infection is not occurring.
“Although we haven’t seen confirmed Zika virus circulation in the continental United States or its territories for several years, it’s still something that we are closely monitoring, particularly as we move into the summer months,” Erin Staples, MD, PhD, medical epidemiologist at the Arboviral Diseases Branch of the Centers for Disease Control and Prevention in Fort Collins, Colo., told this news organization.
“This is because cases are still being reported in other countries, particularly in South America. Travel to these places is increasing following the pandemic, leaving more potential for individuals who might have acquired the infection to come back and restart community transmission.”
How Zika might reemerge
The Aedes aegypti mosquito is the vector by which Zika spreads, and “during the COVID pandemic, these mosquitoes moved further north in the United States, into southern California, and were identified as far north as Washington, D.C.,” said Neil Silverman, MD, professor of clinical obstetrics and gynecology and director of the Infections in Pregnancy Program at UCLA Medical Center in Los Angeles.
“On a population level, Americans have essentially no immunity to Zika from prior infection, and there is no vaccine yet approved. If individuals infected with Zika came into a U.S. region where the Aedes aegypti mosquito was present, that population could be very susceptible to infection spread and even another outbreak. This would be a confluence of bad circumstances, but that’s exactly what infectious disease specialists continue to be watchful about, especially because Zika is so dangerous for fetuses,” said Dr. Silverman.
How the public can prepare
The CDC recommends that pregnant women or women who plan to become pregnant avoid traveling to regions where there are currently outbreaks of Zika, but this is not the only way that individuals can protect themselves.
“The message we want to deliver to people is that in the United States, people are at risk for several mosquito-borne diseases every summer beyond just Zika,” Dr. Staples said. “It’s really important that people are instructed to make a habit of wearing EPA [U.S. Environmental Protection Agency)–registered insect repellents when they go outside. Right now, that is the single best tool that we have to prevent mosquito-borne diseases in the U.S.
“From a community standpoint, there are several emerging mosquito control methods that are being evaluated right now, such as genetic modification and irradiation of mosquitoes. These methods are aimed at producing sterile mosquitoes that are released into the wild to mate with the local mosquito population, which will render them infertile. This leads, over time, to suppression of the overall Aedes aegypti mosquito population – the main vector of Zika transmission,” said Dr. Staples.
Monica Gandhi, MD, MPH, professor of medicine and associate chief of the division of HIV, infectious diseases, and global medicine at the University of California, San Francisco, encourages her patients to wear mosquito repellent but cautioned that “there’s no antiviral that you can take for Zika. Until we have a vaccine, the key to controlling/preventing Zika is controlling the mosquitoes that spread the virus.”
Vaccines
The National Institute of Allergy and Infectious Diseases (NIAID) is currently investigating a variety of Zika vaccines, including a DNA-based vaccine, (phase 2), a purified inactivated virus vaccine (phase 1), live attenuated vaccines (phase 2), and mRNA vaccines (phase 2).
“I’m most excited about mRNA vaccines because they help patients produce a lot of proteins. The protein from a typical protein-adjuvant vaccine will break down, and patients can only raise an immune response to whatever proteins are left. On the other hand, mRNA vaccines provide the body [with] a recipe to make the protein from the pathogen in high amounts, so that a strong immune response can be raised for protection,” noted Dr. Gandhi.
Moderna’s mRNA-1893 vaccine was recently studied in a randomized, observer-blind, controlled, phase 1 trial among 120 adults in the United States and Puerto Rico, the results of which were published online in The Lancet. “The vaccine was found to be generally well tolerated with no serious adverse events considered related to vaccine. Furthermore, the vaccine was able to generate a potent immune response that was capable of neutralizing the virus in vitro,” said Brett Leav, MD, executive director of clinical development for public health vaccines at Moderna.
“Our mRNA platform technology ... can be very helpful against emerging pandemic threats, as we saw in response to COVID-19. What is unique in our approach is that if the genetic sequence of the virus is known, we can quickly generate vaccines to test for their capability to generate a functional immune response. In the case of the mRNA-1893 trial, the vaccine was developed with antigens that were present in the strain of virus circulating in 2016, but we could easily match whatever strain reemerges,” said Dr. Leav.
A phase 2 trial to confirm the dose of mRNA-1893 in a larger study population is underway.
Although it’s been demonstrated that Moderna’s mRNA vaccine is safe and effective, moving from a phase 2 to a phase 3 study presents a challenge, given the fact that currently, the disease burden from Zika is low. If an outbreak were to occur in the future, these mRNA vaccines could potentially be given emergency approval, as occurred during the COVID pandemic, according to Dr. Silverman.
If approved, provisionally or through a traditional route, the vaccine would “accelerate the ability to tamp down any further outbreaks, because vaccine-based immunity could be made available to a large portion of the population who were pregnant or planning a pregnancy, not just in the U.S. but also in these endemic areas,” said Dr. Silverman.
Takeaways from the last Zika outbreak
Practical steps such as mosquito eradication and development of vaccines are not the only takeaway from the recent Zika epidemics inside and outside the United States. A clearer picture of the short- and long-term stakes of the disease has emerged.
According to the CDC, most people who become infected with Zika experience only mild symptoms, such as fever, rash, headache, and muscle pain, but babies conceived by mothers infected with Zika are at risk for stillbirth, miscarriage, and microcephaly and other brain defects.
Although a pregnant woman who tests positive for Zika is in a very high-risk situation, “data show that only about 30% of mothers with Zika have a baby with birth defects. If a pregnant woman contracted Zika, what would happen is we would just do very close screening by ultrasound of the fetus. If microcephaly in utero or fetal brain defects were observed, then a mother would be counseled on her options,” said Dr. Gandhi.
Dr. Silverman noted that “new data on children who were exposed in utero and had normal exams, including head measurements when they were born, have raised concerns. In recently published long-term follow-up studies, even when children born to mothers infected with Zika during pregnancy had normal head growth at least 3 years after birth, they were still at risk for neurodevelopmental delay and behavioral disorders, including impact on coordination and executive function.
“This is another good reason to keep the potential risks of Zika active in the public’s consciousness and in public health planning.”
Dr. Silverman, Dr. Gandhi, and Dr. Staples have disclosed no relevant financial relationships. Dr. Leav is an employee of Moderna and owns stock in the company.
A version of this article originally appeared on Medscape.com.
Warming U.S. temperatures, the resumption of travel, and new knowledge about Zika’s long-term effects on children signal that Zika prevention and vaccine development should be on public health officials’, doctors’, and communities’ radar, even when community infection is not occurring.
“Although we haven’t seen confirmed Zika virus circulation in the continental United States or its territories for several years, it’s still something that we are closely monitoring, particularly as we move into the summer months,” Erin Staples, MD, PhD, medical epidemiologist at the Arboviral Diseases Branch of the Centers for Disease Control and Prevention in Fort Collins, Colo., told this news organization.
“This is because cases are still being reported in other countries, particularly in South America. Travel to these places is increasing following the pandemic, leaving more potential for individuals who might have acquired the infection to come back and restart community transmission.”
How Zika might reemerge
The Aedes aegypti mosquito is the vector by which Zika spreads, and “during the COVID pandemic, these mosquitoes moved further north in the United States, into southern California, and were identified as far north as Washington, D.C.,” said Neil Silverman, MD, professor of clinical obstetrics and gynecology and director of the Infections in Pregnancy Program at UCLA Medical Center in Los Angeles.
“On a population level, Americans have essentially no immunity to Zika from prior infection, and there is no vaccine yet approved. If individuals infected with Zika came into a U.S. region where the Aedes aegypti mosquito was present, that population could be very susceptible to infection spread and even another outbreak. This would be a confluence of bad circumstances, but that’s exactly what infectious disease specialists continue to be watchful about, especially because Zika is so dangerous for fetuses,” said Dr. Silverman.
How the public can prepare
The CDC recommends that pregnant women or women who plan to become pregnant avoid traveling to regions where there are currently outbreaks of Zika, but this is not the only way that individuals can protect themselves.
“The message we want to deliver to people is that in the United States, people are at risk for several mosquito-borne diseases every summer beyond just Zika,” Dr. Staples said. “It’s really important that people are instructed to make a habit of wearing EPA [U.S. Environmental Protection Agency)–registered insect repellents when they go outside. Right now, that is the single best tool that we have to prevent mosquito-borne diseases in the U.S.
“From a community standpoint, there are several emerging mosquito control methods that are being evaluated right now, such as genetic modification and irradiation of mosquitoes. These methods are aimed at producing sterile mosquitoes that are released into the wild to mate with the local mosquito population, which will render them infertile. This leads, over time, to suppression of the overall Aedes aegypti mosquito population – the main vector of Zika transmission,” said Dr. Staples.
Monica Gandhi, MD, MPH, professor of medicine and associate chief of the division of HIV, infectious diseases, and global medicine at the University of California, San Francisco, encourages her patients to wear mosquito repellent but cautioned that “there’s no antiviral that you can take for Zika. Until we have a vaccine, the key to controlling/preventing Zika is controlling the mosquitoes that spread the virus.”
Vaccines
The National Institute of Allergy and Infectious Diseases (NIAID) is currently investigating a variety of Zika vaccines, including a DNA-based vaccine, (phase 2), a purified inactivated virus vaccine (phase 1), live attenuated vaccines (phase 2), and mRNA vaccines (phase 2).
“I’m most excited about mRNA vaccines because they help patients produce a lot of proteins. The protein from a typical protein-adjuvant vaccine will break down, and patients can only raise an immune response to whatever proteins are left. On the other hand, mRNA vaccines provide the body [with] a recipe to make the protein from the pathogen in high amounts, so that a strong immune response can be raised for protection,” noted Dr. Gandhi.
Moderna’s mRNA-1893 vaccine was recently studied in a randomized, observer-blind, controlled, phase 1 trial among 120 adults in the United States and Puerto Rico, the results of which were published online in The Lancet. “The vaccine was found to be generally well tolerated with no serious adverse events considered related to vaccine. Furthermore, the vaccine was able to generate a potent immune response that was capable of neutralizing the virus in vitro,” said Brett Leav, MD, executive director of clinical development for public health vaccines at Moderna.
“Our mRNA platform technology ... can be very helpful against emerging pandemic threats, as we saw in response to COVID-19. What is unique in our approach is that if the genetic sequence of the virus is known, we can quickly generate vaccines to test for their capability to generate a functional immune response. In the case of the mRNA-1893 trial, the vaccine was developed with antigens that were present in the strain of virus circulating in 2016, but we could easily match whatever strain reemerges,” said Dr. Leav.
A phase 2 trial to confirm the dose of mRNA-1893 in a larger study population is underway.
Although it’s been demonstrated that Moderna’s mRNA vaccine is safe and effective, moving from a phase 2 to a phase 3 study presents a challenge, given the fact that currently, the disease burden from Zika is low. If an outbreak were to occur in the future, these mRNA vaccines could potentially be given emergency approval, as occurred during the COVID pandemic, according to Dr. Silverman.
If approved, provisionally or through a traditional route, the vaccine would “accelerate the ability to tamp down any further outbreaks, because vaccine-based immunity could be made available to a large portion of the population who were pregnant or planning a pregnancy, not just in the U.S. but also in these endemic areas,” said Dr. Silverman.
Takeaways from the last Zika outbreak
Practical steps such as mosquito eradication and development of vaccines are not the only takeaway from the recent Zika epidemics inside and outside the United States. A clearer picture of the short- and long-term stakes of the disease has emerged.
According to the CDC, most people who become infected with Zika experience only mild symptoms, such as fever, rash, headache, and muscle pain, but babies conceived by mothers infected with Zika are at risk for stillbirth, miscarriage, and microcephaly and other brain defects.
Although a pregnant woman who tests positive for Zika is in a very high-risk situation, “data show that only about 30% of mothers with Zika have a baby with birth defects. If a pregnant woman contracted Zika, what would happen is we would just do very close screening by ultrasound of the fetus. If microcephaly in utero or fetal brain defects were observed, then a mother would be counseled on her options,” said Dr. Gandhi.
Dr. Silverman noted that “new data on children who were exposed in utero and had normal exams, including head measurements when they were born, have raised concerns. In recently published long-term follow-up studies, even when children born to mothers infected with Zika during pregnancy had normal head growth at least 3 years after birth, they were still at risk for neurodevelopmental delay and behavioral disorders, including impact on coordination and executive function.
“This is another good reason to keep the potential risks of Zika active in the public’s consciousness and in public health planning.”
Dr. Silverman, Dr. Gandhi, and Dr. Staples have disclosed no relevant financial relationships. Dr. Leav is an employee of Moderna and owns stock in the company.
A version of this article originally appeared on Medscape.com.
Warming U.S. temperatures, the resumption of travel, and new knowledge about Zika’s long-term effects on children signal that Zika prevention and vaccine development should be on public health officials’, doctors’, and communities’ radar, even when community infection is not occurring.
“Although we haven’t seen confirmed Zika virus circulation in the continental United States or its territories for several years, it’s still something that we are closely monitoring, particularly as we move into the summer months,” Erin Staples, MD, PhD, medical epidemiologist at the Arboviral Diseases Branch of the Centers for Disease Control and Prevention in Fort Collins, Colo., told this news organization.
“This is because cases are still being reported in other countries, particularly in South America. Travel to these places is increasing following the pandemic, leaving more potential for individuals who might have acquired the infection to come back and restart community transmission.”
How Zika might reemerge
The Aedes aegypti mosquito is the vector by which Zika spreads, and “during the COVID pandemic, these mosquitoes moved further north in the United States, into southern California, and were identified as far north as Washington, D.C.,” said Neil Silverman, MD, professor of clinical obstetrics and gynecology and director of the Infections in Pregnancy Program at UCLA Medical Center in Los Angeles.
“On a population level, Americans have essentially no immunity to Zika from prior infection, and there is no vaccine yet approved. If individuals infected with Zika came into a U.S. region where the Aedes aegypti mosquito was present, that population could be very susceptible to infection spread and even another outbreak. This would be a confluence of bad circumstances, but that’s exactly what infectious disease specialists continue to be watchful about, especially because Zika is so dangerous for fetuses,” said Dr. Silverman.
How the public can prepare
The CDC recommends that pregnant women or women who plan to become pregnant avoid traveling to regions where there are currently outbreaks of Zika, but this is not the only way that individuals can protect themselves.
“The message we want to deliver to people is that in the United States, people are at risk for several mosquito-borne diseases every summer beyond just Zika,” Dr. Staples said. “It’s really important that people are instructed to make a habit of wearing EPA [U.S. Environmental Protection Agency)–registered insect repellents when they go outside. Right now, that is the single best tool that we have to prevent mosquito-borne diseases in the U.S.
“From a community standpoint, there are several emerging mosquito control methods that are being evaluated right now, such as genetic modification and irradiation of mosquitoes. These methods are aimed at producing sterile mosquitoes that are released into the wild to mate with the local mosquito population, which will render them infertile. This leads, over time, to suppression of the overall Aedes aegypti mosquito population – the main vector of Zika transmission,” said Dr. Staples.
Monica Gandhi, MD, MPH, professor of medicine and associate chief of the division of HIV, infectious diseases, and global medicine at the University of California, San Francisco, encourages her patients to wear mosquito repellent but cautioned that “there’s no antiviral that you can take for Zika. Until we have a vaccine, the key to controlling/preventing Zika is controlling the mosquitoes that spread the virus.”
Vaccines
The National Institute of Allergy and Infectious Diseases (NIAID) is currently investigating a variety of Zika vaccines, including a DNA-based vaccine, (phase 2), a purified inactivated virus vaccine (phase 1), live attenuated vaccines (phase 2), and mRNA vaccines (phase 2).
“I’m most excited about mRNA vaccines because they help patients produce a lot of proteins. The protein from a typical protein-adjuvant vaccine will break down, and patients can only raise an immune response to whatever proteins are left. On the other hand, mRNA vaccines provide the body [with] a recipe to make the protein from the pathogen in high amounts, so that a strong immune response can be raised for protection,” noted Dr. Gandhi.
Moderna’s mRNA-1893 vaccine was recently studied in a randomized, observer-blind, controlled, phase 1 trial among 120 adults in the United States and Puerto Rico, the results of which were published online in The Lancet. “The vaccine was found to be generally well tolerated with no serious adverse events considered related to vaccine. Furthermore, the vaccine was able to generate a potent immune response that was capable of neutralizing the virus in vitro,” said Brett Leav, MD, executive director of clinical development for public health vaccines at Moderna.
“Our mRNA platform technology ... can be very helpful against emerging pandemic threats, as we saw in response to COVID-19. What is unique in our approach is that if the genetic sequence of the virus is known, we can quickly generate vaccines to test for their capability to generate a functional immune response. In the case of the mRNA-1893 trial, the vaccine was developed with antigens that were present in the strain of virus circulating in 2016, but we could easily match whatever strain reemerges,” said Dr. Leav.
A phase 2 trial to confirm the dose of mRNA-1893 in a larger study population is underway.
Although it’s been demonstrated that Moderna’s mRNA vaccine is safe and effective, moving from a phase 2 to a phase 3 study presents a challenge, given the fact that currently, the disease burden from Zika is low. If an outbreak were to occur in the future, these mRNA vaccines could potentially be given emergency approval, as occurred during the COVID pandemic, according to Dr. Silverman.
If approved, provisionally or through a traditional route, the vaccine would “accelerate the ability to tamp down any further outbreaks, because vaccine-based immunity could be made available to a large portion of the population who were pregnant or planning a pregnancy, not just in the U.S. but also in these endemic areas,” said Dr. Silverman.
Takeaways from the last Zika outbreak
Practical steps such as mosquito eradication and development of vaccines are not the only takeaway from the recent Zika epidemics inside and outside the United States. A clearer picture of the short- and long-term stakes of the disease has emerged.
According to the CDC, most people who become infected with Zika experience only mild symptoms, such as fever, rash, headache, and muscle pain, but babies conceived by mothers infected with Zika are at risk for stillbirth, miscarriage, and microcephaly and other brain defects.
Although a pregnant woman who tests positive for Zika is in a very high-risk situation, “data show that only about 30% of mothers with Zika have a baby with birth defects. If a pregnant woman contracted Zika, what would happen is we would just do very close screening by ultrasound of the fetus. If microcephaly in utero or fetal brain defects were observed, then a mother would be counseled on her options,” said Dr. Gandhi.
Dr. Silverman noted that “new data on children who were exposed in utero and had normal exams, including head measurements when they were born, have raised concerns. In recently published long-term follow-up studies, even when children born to mothers infected with Zika during pregnancy had normal head growth at least 3 years after birth, they were still at risk for neurodevelopmental delay and behavioral disorders, including impact on coordination and executive function.
“This is another good reason to keep the potential risks of Zika active in the public’s consciousness and in public health planning.”
Dr. Silverman, Dr. Gandhi, and Dr. Staples have disclosed no relevant financial relationships. Dr. Leav is an employee of Moderna and owns stock in the company.
A version of this article originally appeared on Medscape.com.
FDA OKs elacestrant for ESR1+ advanced, metastatic breast cancer
that progressed on at least one line of endocrine therapy.
The agency also approved the Guardant360 CDx assay as a companion diagnostic to identify breast cancer patients who meet the treatment requirements, according to the agency’s press release announcing the approval.
The novel oral selective estrogen receptor degrader was approved based on the phase 3 EMERALD trial, which included 478 postmenopausal women and men with ER-positive, HER2-negative advanced or metastatic breast cancer, about half of whom had ESR1 mutations. Patients had progressed on one or two prior lines of endocrine therapy, including one containing a CDK4/6 inhibitor. Participants could also have had one prior line of chemotherapy in the advanced or metastatic setting.
Participants were randomized 1:1 to either elacestrant 345 mg orally once daily or investigator’s choice of endocrine therapy, which included fulvestrant or an aromatase inhibitor.
In the 228 patients (48%) with ESR1 mutations, median progression-free survival (PFS) was 3.8 months with elacestrant versus 1.9 months in the fulvestrant or aromatase inhibitor arm (hazard ratio, 0.55; P = .0005). Investigators observed no statistically significant PFS difference between the treatment arms in patients who didn’t have the mutation.
Fair comparison?
In June, experts raised concerns about the adequacy of the “standard of care” control arm in EMERALD, particularly that single agents were used at a time when combination therapy is becoming more common.
“The expression ‘standard of care’ is applied generously, as the control arm is restricted” to single agents and no combinations, which “may have led to a substandard” comparison group, Timothée Olivier, MD, Geneva University Hospital, and Vinay Prasad, MD, MPH, University of California, San Francisco, said in an editorial quoted in the piece.
EMERALD investigators acknowledged that there were issues with the control group, noting that in the “United States and Europe, combination therapy with fulvestrant” – instead of single agents – “is increasingly being used as the second-line [standard of care] treatment.”
However, the goal of the study “was to compare a novel endocrine therapy vs. currently available endocrine therapies,” not combination regimens, the investigators said.
Also, “the benefit of elacestrant over fulvestrant and AIs [aromatase inhibitors] in our monotherapy trial ... suggests that incorporating elacestrant as the preferred endocrine therapy backbone in future earlier-line combination studies is a promising strategy.”
Lipid monitoring necessary
The most common adverse events with elacestrant, occurring in 10% or more of patients, are musculoskeletal pain, nausea, increased cholesterol, increased AST, increased triglycerides, fatigue, decreased hemoglobin, vomiting, increased ALT, decreased sodium, increased creatinine, decreased appetite, diarrhea, headache, constipation, abdominal pain, hot flush, and dyspepsia, according to labeling.
Labeling warns that elacestrant “may cause hypercholesterolemia and hypertriglyceridemia. Monitor lipid profile prior to starting treatment and periodically thereafter.”
The recommended elacestrant dose is the trial dose, 345 mg orally with food once daily until disease progression or unacceptable toxicity.
A version of this article first appeared on Medscape.com.
that progressed on at least one line of endocrine therapy.
The agency also approved the Guardant360 CDx assay as a companion diagnostic to identify breast cancer patients who meet the treatment requirements, according to the agency’s press release announcing the approval.
The novel oral selective estrogen receptor degrader was approved based on the phase 3 EMERALD trial, which included 478 postmenopausal women and men with ER-positive, HER2-negative advanced or metastatic breast cancer, about half of whom had ESR1 mutations. Patients had progressed on one or two prior lines of endocrine therapy, including one containing a CDK4/6 inhibitor. Participants could also have had one prior line of chemotherapy in the advanced or metastatic setting.
Participants were randomized 1:1 to either elacestrant 345 mg orally once daily or investigator’s choice of endocrine therapy, which included fulvestrant or an aromatase inhibitor.
In the 228 patients (48%) with ESR1 mutations, median progression-free survival (PFS) was 3.8 months with elacestrant versus 1.9 months in the fulvestrant or aromatase inhibitor arm (hazard ratio, 0.55; P = .0005). Investigators observed no statistically significant PFS difference between the treatment arms in patients who didn’t have the mutation.
Fair comparison?
In June, experts raised concerns about the adequacy of the “standard of care” control arm in EMERALD, particularly that single agents were used at a time when combination therapy is becoming more common.
“The expression ‘standard of care’ is applied generously, as the control arm is restricted” to single agents and no combinations, which “may have led to a substandard” comparison group, Timothée Olivier, MD, Geneva University Hospital, and Vinay Prasad, MD, MPH, University of California, San Francisco, said in an editorial quoted in the piece.
EMERALD investigators acknowledged that there were issues with the control group, noting that in the “United States and Europe, combination therapy with fulvestrant” – instead of single agents – “is increasingly being used as the second-line [standard of care] treatment.”
However, the goal of the study “was to compare a novel endocrine therapy vs. currently available endocrine therapies,” not combination regimens, the investigators said.
Also, “the benefit of elacestrant over fulvestrant and AIs [aromatase inhibitors] in our monotherapy trial ... suggests that incorporating elacestrant as the preferred endocrine therapy backbone in future earlier-line combination studies is a promising strategy.”
Lipid monitoring necessary
The most common adverse events with elacestrant, occurring in 10% or more of patients, are musculoskeletal pain, nausea, increased cholesterol, increased AST, increased triglycerides, fatigue, decreased hemoglobin, vomiting, increased ALT, decreased sodium, increased creatinine, decreased appetite, diarrhea, headache, constipation, abdominal pain, hot flush, and dyspepsia, according to labeling.
Labeling warns that elacestrant “may cause hypercholesterolemia and hypertriglyceridemia. Monitor lipid profile prior to starting treatment and periodically thereafter.”
The recommended elacestrant dose is the trial dose, 345 mg orally with food once daily until disease progression or unacceptable toxicity.
A version of this article first appeared on Medscape.com.
that progressed on at least one line of endocrine therapy.
The agency also approved the Guardant360 CDx assay as a companion diagnostic to identify breast cancer patients who meet the treatment requirements, according to the agency’s press release announcing the approval.
The novel oral selective estrogen receptor degrader was approved based on the phase 3 EMERALD trial, which included 478 postmenopausal women and men with ER-positive, HER2-negative advanced or metastatic breast cancer, about half of whom had ESR1 mutations. Patients had progressed on one or two prior lines of endocrine therapy, including one containing a CDK4/6 inhibitor. Participants could also have had one prior line of chemotherapy in the advanced or metastatic setting.
Participants were randomized 1:1 to either elacestrant 345 mg orally once daily or investigator’s choice of endocrine therapy, which included fulvestrant or an aromatase inhibitor.
In the 228 patients (48%) with ESR1 mutations, median progression-free survival (PFS) was 3.8 months with elacestrant versus 1.9 months in the fulvestrant or aromatase inhibitor arm (hazard ratio, 0.55; P = .0005). Investigators observed no statistically significant PFS difference between the treatment arms in patients who didn’t have the mutation.
Fair comparison?
In June, experts raised concerns about the adequacy of the “standard of care” control arm in EMERALD, particularly that single agents were used at a time when combination therapy is becoming more common.
“The expression ‘standard of care’ is applied generously, as the control arm is restricted” to single agents and no combinations, which “may have led to a substandard” comparison group, Timothée Olivier, MD, Geneva University Hospital, and Vinay Prasad, MD, MPH, University of California, San Francisco, said in an editorial quoted in the piece.
EMERALD investigators acknowledged that there were issues with the control group, noting that in the “United States and Europe, combination therapy with fulvestrant” – instead of single agents – “is increasingly being used as the second-line [standard of care] treatment.”
However, the goal of the study “was to compare a novel endocrine therapy vs. currently available endocrine therapies,” not combination regimens, the investigators said.
Also, “the benefit of elacestrant over fulvestrant and AIs [aromatase inhibitors] in our monotherapy trial ... suggests that incorporating elacestrant as the preferred endocrine therapy backbone in future earlier-line combination studies is a promising strategy.”
Lipid monitoring necessary
The most common adverse events with elacestrant, occurring in 10% or more of patients, are musculoskeletal pain, nausea, increased cholesterol, increased AST, increased triglycerides, fatigue, decreased hemoglobin, vomiting, increased ALT, decreased sodium, increased creatinine, decreased appetite, diarrhea, headache, constipation, abdominal pain, hot flush, and dyspepsia, according to labeling.
Labeling warns that elacestrant “may cause hypercholesterolemia and hypertriglyceridemia. Monitor lipid profile prior to starting treatment and periodically thereafter.”
The recommended elacestrant dose is the trial dose, 345 mg orally with food once daily until disease progression or unacceptable toxicity.
A version of this article first appeared on Medscape.com.
First Humira biosimilar launches in U.S.
The first biosimilar for Humira, adalimumab-atto (Amjevita), is now available in the United States, according to an announcement on Jan. 31 by the manufacturer, Amgen. At least seven other U.S. Food and Drug Administration–approved Humira biosimilars are expected to become available later in 2023.
Amjevita was approved by the FDA in September 2016 for multiple inflammatory diseases, including rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, and plaque psoriasis. The delayed launch was part of a global settlement with Humira’s manufacturer, AbbVie.
Humira (adalimumab) has been available since 2002 and is consistently one of the top-selling drugs in the United States. A single 40-mg Amjevita pen device will be available at two prices: a list price (wholesale acquisition cost) of $1,557.59, 55% below the current Humira list price, and a list price of $3,288.24, 5% below the current Humira list price, according to Amgen.
“Amgen’s goal is to provide broad access for patients by offering two options to health plans and pharmacy benefit managers,” the company said in the press release.
Patients are less likely to benefit from the more significant discount, said Marta Wosinska, PhD, a health care economist at the Brookings Institute in Washington, DC. It's expected that insurance companies will use the higher list price for Amjevita, she said, as this higher price will also likely have higher rebates. Rebates are payments to health insurance payers provided by drug manufacturers to promote use of an expensive drug. Some pharmacy benefit managers have already said that they plan to charge patients the same amount for Humira as its biosimilars, Dr. Wosinska said.
"For an existing patient, there's really no incentive for them to switch," she said in an interview.
So far only one insurance company, Kaiser Permanente, has plans to switch patients over to biosimilars, according to the health policy podcast Tradeoffs, and the insurer will stop covering Humira by the end of this year.
A version of this article first appeared on Medscape.com.
*This story was updated 2/1/2023.
The first biosimilar for Humira, adalimumab-atto (Amjevita), is now available in the United States, according to an announcement on Jan. 31 by the manufacturer, Amgen. At least seven other U.S. Food and Drug Administration–approved Humira biosimilars are expected to become available later in 2023.
Amjevita was approved by the FDA in September 2016 for multiple inflammatory diseases, including rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, and plaque psoriasis. The delayed launch was part of a global settlement with Humira’s manufacturer, AbbVie.
Humira (adalimumab) has been available since 2002 and is consistently one of the top-selling drugs in the United States. A single 40-mg Amjevita pen device will be available at two prices: a list price (wholesale acquisition cost) of $1,557.59, 55% below the current Humira list price, and a list price of $3,288.24, 5% below the current Humira list price, according to Amgen.
“Amgen’s goal is to provide broad access for patients by offering two options to health plans and pharmacy benefit managers,” the company said in the press release.
Patients are less likely to benefit from the more significant discount, said Marta Wosinska, PhD, a health care economist at the Brookings Institute in Washington, DC. It's expected that insurance companies will use the higher list price for Amjevita, she said, as this higher price will also likely have higher rebates. Rebates are payments to health insurance payers provided by drug manufacturers to promote use of an expensive drug. Some pharmacy benefit managers have already said that they plan to charge patients the same amount for Humira as its biosimilars, Dr. Wosinska said.
"For an existing patient, there's really no incentive for them to switch," she said in an interview.
So far only one insurance company, Kaiser Permanente, has plans to switch patients over to biosimilars, according to the health policy podcast Tradeoffs, and the insurer will stop covering Humira by the end of this year.
A version of this article first appeared on Medscape.com.
*This story was updated 2/1/2023.
The first biosimilar for Humira, adalimumab-atto (Amjevita), is now available in the United States, according to an announcement on Jan. 31 by the manufacturer, Amgen. At least seven other U.S. Food and Drug Administration–approved Humira biosimilars are expected to become available later in 2023.
Amjevita was approved by the FDA in September 2016 for multiple inflammatory diseases, including rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, and plaque psoriasis. The delayed launch was part of a global settlement with Humira’s manufacturer, AbbVie.
Humira (adalimumab) has been available since 2002 and is consistently one of the top-selling drugs in the United States. A single 40-mg Amjevita pen device will be available at two prices: a list price (wholesale acquisition cost) of $1,557.59, 55% below the current Humira list price, and a list price of $3,288.24, 5% below the current Humira list price, according to Amgen.
“Amgen’s goal is to provide broad access for patients by offering two options to health plans and pharmacy benefit managers,” the company said in the press release.
Patients are less likely to benefit from the more significant discount, said Marta Wosinska, PhD, a health care economist at the Brookings Institute in Washington, DC. It's expected that insurance companies will use the higher list price for Amjevita, she said, as this higher price will also likely have higher rebates. Rebates are payments to health insurance payers provided by drug manufacturers to promote use of an expensive drug. Some pharmacy benefit managers have already said that they plan to charge patients the same amount for Humira as its biosimilars, Dr. Wosinska said.
"For an existing patient, there's really no incentive for them to switch," she said in an interview.
So far only one insurance company, Kaiser Permanente, has plans to switch patients over to biosimilars, according to the health policy podcast Tradeoffs, and the insurer will stop covering Humira by the end of this year.
A version of this article first appeared on Medscape.com.
*This story was updated 2/1/2023.
FDA approves pirtobrutinib for r/r mantle cell lymphoma
Pirtobrutinib is the first and only noncovalent Bruton’s tyrosine kinase inhibitor approved for use in this MCL setting, manufacturer Eli Lilly noted in a press release.
“The approval of Jaypirca represents an important advance for patients with relapsed or refractory MCL, who currently have limited options and historically have had a poor prognosis following discontinuation of treatment with a covalent Bruton’s tyrosine kinase inhibitor,” senior author Michael Wang, MD, University of Texas MD Anderson Cancer Center, Houston, said in the release.
The approval was based on efficacy demonstrated in the open-label, single-arm, phase 1/2 BRUIN trial – a multicenter study assessing 200 mg once-daily oral pirtobrutinib monotherapy in 120 patients with MCL who had previously received a Bruton’s tyrosine kinase inhibitor, most often ibrutinib (Imbruvica, 67%) acalabrutinib (Calquence, 30%) and zanubrutinib (Brukinsa, 8%). Pirtobrutinib was continued until disease progression or unacceptable toxicity.
Study participants had a median of three prior lines of therapy, and 83% discontinued their last Bruton’s tyrosine kinase inhibitor because of refractory or progressive disease.
The overall response rate in pirtobrutinib-treated patients was 50% with a complete response rate of 13%. Estimated median duration of response was 8.3 months, and the estimated duration of response at 6 months occurred in nearly two-thirds of patients.
Adverse reactions that occurred in at least 15% of patients included fatigue, musculoskeletal pain, diarrhea, edema, dyspnea, pneumonia, and bruising. Grade 3 or 4 laboratory abnormalities occurring in at least 10% of patients included decreased neutrophil counts, lymphocyte counts, and platelet counts.
Prescribing information for pirtobrutinib includes warnings and precautions for infections, hemorrhage, cytopenias, atrial fibrillation and flutter, and second primary malignancies, noted the FDA, which granted priority review, fast track designation, and orphan drug designation for the application submitted by Eli Lilly.
“Jaypirca can reestablish Bruton’s tyrosine kinase inhibition in MCL patients previously treated with a covalent Bruton’s tyrosine kinase inhibitor (ibrutinib, acalabrutinib, or zanubrutinib) and extend the benefit of targeting the Bruton’s tyrosine kinase pathway,” according to Eli Lilly’s release.
Dr. Wang added that the agent “has the potential to meaningfully impact the treatment paradigm for relapsed and refractory MCL patients.”
Meghan Gutierrez, CEO at the Lymphoma Research Foundation, also noted that “the approval of Jaypirca brings a new treatment option and, along with that, new hope for people with relapsed or refractory MCL.”
The drug is expected to be available in the United States in the coming weeks, and the confirmatory phase 3 BRUIN trial is currently enrolling patients, Eli Lilly announced. The company also indicated the list price would be $21,000 for a 30-day supply of the 200-mg dose.
Serious adverse events believed to be associated with the use of pirtobrutinib or any medicine or device should be reported to the FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.
A version of this article first appeared on Medscape.com.
Pirtobrutinib is the first and only noncovalent Bruton’s tyrosine kinase inhibitor approved for use in this MCL setting, manufacturer Eli Lilly noted in a press release.
“The approval of Jaypirca represents an important advance for patients with relapsed or refractory MCL, who currently have limited options and historically have had a poor prognosis following discontinuation of treatment with a covalent Bruton’s tyrosine kinase inhibitor,” senior author Michael Wang, MD, University of Texas MD Anderson Cancer Center, Houston, said in the release.
The approval was based on efficacy demonstrated in the open-label, single-arm, phase 1/2 BRUIN trial – a multicenter study assessing 200 mg once-daily oral pirtobrutinib monotherapy in 120 patients with MCL who had previously received a Bruton’s tyrosine kinase inhibitor, most often ibrutinib (Imbruvica, 67%) acalabrutinib (Calquence, 30%) and zanubrutinib (Brukinsa, 8%). Pirtobrutinib was continued until disease progression or unacceptable toxicity.
Study participants had a median of three prior lines of therapy, and 83% discontinued their last Bruton’s tyrosine kinase inhibitor because of refractory or progressive disease.
The overall response rate in pirtobrutinib-treated patients was 50% with a complete response rate of 13%. Estimated median duration of response was 8.3 months, and the estimated duration of response at 6 months occurred in nearly two-thirds of patients.
Adverse reactions that occurred in at least 15% of patients included fatigue, musculoskeletal pain, diarrhea, edema, dyspnea, pneumonia, and bruising. Grade 3 or 4 laboratory abnormalities occurring in at least 10% of patients included decreased neutrophil counts, lymphocyte counts, and platelet counts.
Prescribing information for pirtobrutinib includes warnings and precautions for infections, hemorrhage, cytopenias, atrial fibrillation and flutter, and second primary malignancies, noted the FDA, which granted priority review, fast track designation, and orphan drug designation for the application submitted by Eli Lilly.
“Jaypirca can reestablish Bruton’s tyrosine kinase inhibition in MCL patients previously treated with a covalent Bruton’s tyrosine kinase inhibitor (ibrutinib, acalabrutinib, or zanubrutinib) and extend the benefit of targeting the Bruton’s tyrosine kinase pathway,” according to Eli Lilly’s release.
Dr. Wang added that the agent “has the potential to meaningfully impact the treatment paradigm for relapsed and refractory MCL patients.”
Meghan Gutierrez, CEO at the Lymphoma Research Foundation, also noted that “the approval of Jaypirca brings a new treatment option and, along with that, new hope for people with relapsed or refractory MCL.”
The drug is expected to be available in the United States in the coming weeks, and the confirmatory phase 3 BRUIN trial is currently enrolling patients, Eli Lilly announced. The company also indicated the list price would be $21,000 for a 30-day supply of the 200-mg dose.
Serious adverse events believed to be associated with the use of pirtobrutinib or any medicine or device should be reported to the FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.
A version of this article first appeared on Medscape.com.
Pirtobrutinib is the first and only noncovalent Bruton’s tyrosine kinase inhibitor approved for use in this MCL setting, manufacturer Eli Lilly noted in a press release.
“The approval of Jaypirca represents an important advance for patients with relapsed or refractory MCL, who currently have limited options and historically have had a poor prognosis following discontinuation of treatment with a covalent Bruton’s tyrosine kinase inhibitor,” senior author Michael Wang, MD, University of Texas MD Anderson Cancer Center, Houston, said in the release.
The approval was based on efficacy demonstrated in the open-label, single-arm, phase 1/2 BRUIN trial – a multicenter study assessing 200 mg once-daily oral pirtobrutinib monotherapy in 120 patients with MCL who had previously received a Bruton’s tyrosine kinase inhibitor, most often ibrutinib (Imbruvica, 67%) acalabrutinib (Calquence, 30%) and zanubrutinib (Brukinsa, 8%). Pirtobrutinib was continued until disease progression or unacceptable toxicity.
Study participants had a median of three prior lines of therapy, and 83% discontinued their last Bruton’s tyrosine kinase inhibitor because of refractory or progressive disease.
The overall response rate in pirtobrutinib-treated patients was 50% with a complete response rate of 13%. Estimated median duration of response was 8.3 months, and the estimated duration of response at 6 months occurred in nearly two-thirds of patients.
Adverse reactions that occurred in at least 15% of patients included fatigue, musculoskeletal pain, diarrhea, edema, dyspnea, pneumonia, and bruising. Grade 3 or 4 laboratory abnormalities occurring in at least 10% of patients included decreased neutrophil counts, lymphocyte counts, and platelet counts.
Prescribing information for pirtobrutinib includes warnings and precautions for infections, hemorrhage, cytopenias, atrial fibrillation and flutter, and second primary malignancies, noted the FDA, which granted priority review, fast track designation, and orphan drug designation for the application submitted by Eli Lilly.
“Jaypirca can reestablish Bruton’s tyrosine kinase inhibition in MCL patients previously treated with a covalent Bruton’s tyrosine kinase inhibitor (ibrutinib, acalabrutinib, or zanubrutinib) and extend the benefit of targeting the Bruton’s tyrosine kinase pathway,” according to Eli Lilly’s release.
Dr. Wang added that the agent “has the potential to meaningfully impact the treatment paradigm for relapsed and refractory MCL patients.”
Meghan Gutierrez, CEO at the Lymphoma Research Foundation, also noted that “the approval of Jaypirca brings a new treatment option and, along with that, new hope for people with relapsed or refractory MCL.”
The drug is expected to be available in the United States in the coming weeks, and the confirmatory phase 3 BRUIN trial is currently enrolling patients, Eli Lilly announced. The company also indicated the list price would be $21,000 for a 30-day supply of the 200-mg dose.
Serious adverse events believed to be associated with the use of pirtobrutinib or any medicine or device should be reported to the FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.
A version of this article first appeared on Medscape.com.
FDA panel backs shift toward one-dose COVID shot
The FDA is looking to give clearer direction to vaccine makers about future development of COVID-19 vaccines. The plan is to narrow down the current complex landscape of options for vaccinations, and thus help increase use of these shots.
COVID remains a serious threat, causing about 4,000 deaths a week recently, according to the Centers for Disease Control and Prevention. The 21 members of the Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Jan. 26 voted unanimously “yes” on a single question posed by the FDA:
“Does the committee recommend harmonizing the vaccine strain composition of primary series and booster doses in the U.S. to a single composition, e.g., the composition for all vaccines administered currently would be a bivalent vaccine (Original plus Omicron BA.4/BA.5)?”
In other words, would it be better to have one vaccine potentially combining multiple strains of the virus, instead of multiple vaccines – such as a two-shot primary series then a booster containing different combinations of viral strains.
The FDA will consider the panel’s advice as it outlines new strategies for keeping ahead of the evolving virus.
In explaining their support for the FDA plan, panel members said they hoped that a simpler regime would aid in persuading more people to get COVID vaccines.
Pamela McInnes, DDS, MSc, noted that it’s difficult to explain to many people that the vaccine works to protect them from more severe illness if they contract COVID after getting vaccinated.
“That is a real challenge,” said Dr. McInness, retired deputy director of the National Center for Advancing Translational Sciences at the National Institutes of Health.
“The message that you would have gotten more sick and landed in the hospital resonates with me, but I’m not sure if it resonates with” many people who become infected, she said.
The plan
In the briefing document for the meeting, the FDA outlined a plan for transitioning from the current complex landscape of COVID-19 vaccines to a single vaccine composition for the primary series and booster vaccination.
This would require harmonizing the strain composition of all COVID-19 vaccines; simplifying the immunization schedule for future vaccination campaigns to administer a two-dose series in certain young children and in older adults and persons with compromised immunity, and only one dose in all other individuals; and establishing a process for vaccine strain selection recommendations, similar in many ways to that used for seasonal influenza vaccines, based on prevailing and predicted variants that would take place by June to allow for vaccine production by September.
During the discussion, though, questions arose about the June target date. Given the production schedule for some vaccines, that date might need to shift, said Jerry Weir, PhD, director of the division of viral products at FDA’s Center for Biologics Evaluation and Research.
“We’re all just going to have to maintain flexibility,” Dr. Weir said, adding that there is not yet a “good pattern” established for updating these vaccines.
Increasing vaccination rates
There was broad consensus about the need to boost public support for COVID-19 vaccinations. While about 81% of the U.S. population has had at least one dose of this vaccine, only 15.3% have had an updated bivalent booster dose, according to the CDC.
“Anything that results in better public communication would be extremely valuable,” said committee member Henry H. Bernstein, DO, MHCM, of the Zucker School of Medicine at Hofstra/Northwell Health in Hempstead, N.Y.
But it’s unclear what expectations will be prioritized for the COVID vaccine program, he said.
“Realistically, I don’t think we can have it all – less infection, less transmission, less severe disease, and less long COVID,” Dr. Bernstein said. “And that seems to be a major challenge for public messaging.”
Panelists press for more data
Other committee members also pressed for clearer targets in evaluating the goals for COVID vaccines, and for more robust data.
Like his fellow VRBPAC members, Cody Meissner, MD, of Dartmouth’s Geisel School of Medicine, Hanover, N.H., supported a move toward harmonizing the strains used in different companies’ vaccines. But he added that it wasn’t clear yet how frequently they should be administered.
“We need to see what happens with disease burden,” Dr. Meissner said. “We may or may not need annual vaccination. It’s just awfully early, it seems to me, in this process to answer that question.”
Among those serving on VRBPAC was one of the FDA’s more vocal critics on these points, Paul A. Offit, MD, a vaccine expert from Children’s Hospital of Philadelphia. Dr. Offit, for example, joined former FDA officials in writing a November opinion article for the Washington Post, arguing that the evidence for boosters for healthy younger adults was not strong.
At the Jan. 26 meeting, he supported the drive toward simplification of COVID vaccine schedules, while arguing for more data about how well these products are working.
“This virus is going to be with us for years, if not decades, and there will always be vulnerable groups who are going to be hospitalized and killed by the virus,” Dr. Offit said.
The CDC needs to provide more information about the characteristics of people being hospitalized with COVID infections, including their ages and comorbidities as well as details about their vaccine history, he said. In addition, academic researchers should provide a clearer picture of what immunological predictors are at play in increasing people’s risk from COVID.
“Then and only then can we really best make the decision about who gets vaccinated with what and when,” Dr. Offit said.
VRBPAC member Ofer Levy, MD, PhD, also urged the FDA to press for a collection of more robust and detailed information about the immune response to COVID-19 vaccinations, such as a deeper look at what’s happening with antibodies.
“I hope FDA will continue to reflect on how to best take this information forward, and encourage – or require – sponsors to gather more information in a standardized way across these different arms of the human immune system,” Dr. Levy said. “So we keep learning and keep doing this better.”
In recapping the panel’s suggestions at the end of the meeting, Peter Marks, MD, PhD, the director of the FDA’s Center for Biologics Evaluation and Research, addressed the requests made during the day’s meeting about better data on how the vaccines work.
“We heard loud and clear that we need to use a data-driven approach to get to the simplest possible scheme that we can for vaccination,” Dr. Marks said. “And it should be as simple as possible but not oversimplified, a little bit like they say about Mozart’s music.”
A version of this article first appeared on WebMD.com.
The FDA is looking to give clearer direction to vaccine makers about future development of COVID-19 vaccines. The plan is to narrow down the current complex landscape of options for vaccinations, and thus help increase use of these shots.
COVID remains a serious threat, causing about 4,000 deaths a week recently, according to the Centers for Disease Control and Prevention. The 21 members of the Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Jan. 26 voted unanimously “yes” on a single question posed by the FDA:
“Does the committee recommend harmonizing the vaccine strain composition of primary series and booster doses in the U.S. to a single composition, e.g., the composition for all vaccines administered currently would be a bivalent vaccine (Original plus Omicron BA.4/BA.5)?”
In other words, would it be better to have one vaccine potentially combining multiple strains of the virus, instead of multiple vaccines – such as a two-shot primary series then a booster containing different combinations of viral strains.
The FDA will consider the panel’s advice as it outlines new strategies for keeping ahead of the evolving virus.
In explaining their support for the FDA plan, panel members said they hoped that a simpler regime would aid in persuading more people to get COVID vaccines.
Pamela McInnes, DDS, MSc, noted that it’s difficult to explain to many people that the vaccine works to protect them from more severe illness if they contract COVID after getting vaccinated.
“That is a real challenge,” said Dr. McInness, retired deputy director of the National Center for Advancing Translational Sciences at the National Institutes of Health.
“The message that you would have gotten more sick and landed in the hospital resonates with me, but I’m not sure if it resonates with” many people who become infected, she said.
The plan
In the briefing document for the meeting, the FDA outlined a plan for transitioning from the current complex landscape of COVID-19 vaccines to a single vaccine composition for the primary series and booster vaccination.
This would require harmonizing the strain composition of all COVID-19 vaccines; simplifying the immunization schedule for future vaccination campaigns to administer a two-dose series in certain young children and in older adults and persons with compromised immunity, and only one dose in all other individuals; and establishing a process for vaccine strain selection recommendations, similar in many ways to that used for seasonal influenza vaccines, based on prevailing and predicted variants that would take place by June to allow for vaccine production by September.
During the discussion, though, questions arose about the June target date. Given the production schedule for some vaccines, that date might need to shift, said Jerry Weir, PhD, director of the division of viral products at FDA’s Center for Biologics Evaluation and Research.
“We’re all just going to have to maintain flexibility,” Dr. Weir said, adding that there is not yet a “good pattern” established for updating these vaccines.
Increasing vaccination rates
There was broad consensus about the need to boost public support for COVID-19 vaccinations. While about 81% of the U.S. population has had at least one dose of this vaccine, only 15.3% have had an updated bivalent booster dose, according to the CDC.
“Anything that results in better public communication would be extremely valuable,” said committee member Henry H. Bernstein, DO, MHCM, of the Zucker School of Medicine at Hofstra/Northwell Health in Hempstead, N.Y.
But it’s unclear what expectations will be prioritized for the COVID vaccine program, he said.
“Realistically, I don’t think we can have it all – less infection, less transmission, less severe disease, and less long COVID,” Dr. Bernstein said. “And that seems to be a major challenge for public messaging.”
Panelists press for more data
Other committee members also pressed for clearer targets in evaluating the goals for COVID vaccines, and for more robust data.
Like his fellow VRBPAC members, Cody Meissner, MD, of Dartmouth’s Geisel School of Medicine, Hanover, N.H., supported a move toward harmonizing the strains used in different companies’ vaccines. But he added that it wasn’t clear yet how frequently they should be administered.
“We need to see what happens with disease burden,” Dr. Meissner said. “We may or may not need annual vaccination. It’s just awfully early, it seems to me, in this process to answer that question.”
Among those serving on VRBPAC was one of the FDA’s more vocal critics on these points, Paul A. Offit, MD, a vaccine expert from Children’s Hospital of Philadelphia. Dr. Offit, for example, joined former FDA officials in writing a November opinion article for the Washington Post, arguing that the evidence for boosters for healthy younger adults was not strong.
At the Jan. 26 meeting, he supported the drive toward simplification of COVID vaccine schedules, while arguing for more data about how well these products are working.
“This virus is going to be with us for years, if not decades, and there will always be vulnerable groups who are going to be hospitalized and killed by the virus,” Dr. Offit said.
The CDC needs to provide more information about the characteristics of people being hospitalized with COVID infections, including their ages and comorbidities as well as details about their vaccine history, he said. In addition, academic researchers should provide a clearer picture of what immunological predictors are at play in increasing people’s risk from COVID.
“Then and only then can we really best make the decision about who gets vaccinated with what and when,” Dr. Offit said.
VRBPAC member Ofer Levy, MD, PhD, also urged the FDA to press for a collection of more robust and detailed information about the immune response to COVID-19 vaccinations, such as a deeper look at what’s happening with antibodies.
“I hope FDA will continue to reflect on how to best take this information forward, and encourage – or require – sponsors to gather more information in a standardized way across these different arms of the human immune system,” Dr. Levy said. “So we keep learning and keep doing this better.”
In recapping the panel’s suggestions at the end of the meeting, Peter Marks, MD, PhD, the director of the FDA’s Center for Biologics Evaluation and Research, addressed the requests made during the day’s meeting about better data on how the vaccines work.
“We heard loud and clear that we need to use a data-driven approach to get to the simplest possible scheme that we can for vaccination,” Dr. Marks said. “And it should be as simple as possible but not oversimplified, a little bit like they say about Mozart’s music.”
A version of this article first appeared on WebMD.com.
The FDA is looking to give clearer direction to vaccine makers about future development of COVID-19 vaccines. The plan is to narrow down the current complex landscape of options for vaccinations, and thus help increase use of these shots.
COVID remains a serious threat, causing about 4,000 deaths a week recently, according to the Centers for Disease Control and Prevention. The 21 members of the Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Jan. 26 voted unanimously “yes” on a single question posed by the FDA:
“Does the committee recommend harmonizing the vaccine strain composition of primary series and booster doses in the U.S. to a single composition, e.g., the composition for all vaccines administered currently would be a bivalent vaccine (Original plus Omicron BA.4/BA.5)?”
In other words, would it be better to have one vaccine potentially combining multiple strains of the virus, instead of multiple vaccines – such as a two-shot primary series then a booster containing different combinations of viral strains.
The FDA will consider the panel’s advice as it outlines new strategies for keeping ahead of the evolving virus.
In explaining their support for the FDA plan, panel members said they hoped that a simpler regime would aid in persuading more people to get COVID vaccines.
Pamela McInnes, DDS, MSc, noted that it’s difficult to explain to many people that the vaccine works to protect them from more severe illness if they contract COVID after getting vaccinated.
“That is a real challenge,” said Dr. McInness, retired deputy director of the National Center for Advancing Translational Sciences at the National Institutes of Health.
“The message that you would have gotten more sick and landed in the hospital resonates with me, but I’m not sure if it resonates with” many people who become infected, she said.
The plan
In the briefing document for the meeting, the FDA outlined a plan for transitioning from the current complex landscape of COVID-19 vaccines to a single vaccine composition for the primary series and booster vaccination.
This would require harmonizing the strain composition of all COVID-19 vaccines; simplifying the immunization schedule for future vaccination campaigns to administer a two-dose series in certain young children and in older adults and persons with compromised immunity, and only one dose in all other individuals; and establishing a process for vaccine strain selection recommendations, similar in many ways to that used for seasonal influenza vaccines, based on prevailing and predicted variants that would take place by June to allow for vaccine production by September.
During the discussion, though, questions arose about the June target date. Given the production schedule for some vaccines, that date might need to shift, said Jerry Weir, PhD, director of the division of viral products at FDA’s Center for Biologics Evaluation and Research.
“We’re all just going to have to maintain flexibility,” Dr. Weir said, adding that there is not yet a “good pattern” established for updating these vaccines.
Increasing vaccination rates
There was broad consensus about the need to boost public support for COVID-19 vaccinations. While about 81% of the U.S. population has had at least one dose of this vaccine, only 15.3% have had an updated bivalent booster dose, according to the CDC.
“Anything that results in better public communication would be extremely valuable,” said committee member Henry H. Bernstein, DO, MHCM, of the Zucker School of Medicine at Hofstra/Northwell Health in Hempstead, N.Y.
But it’s unclear what expectations will be prioritized for the COVID vaccine program, he said.
“Realistically, I don’t think we can have it all – less infection, less transmission, less severe disease, and less long COVID,” Dr. Bernstein said. “And that seems to be a major challenge for public messaging.”
Panelists press for more data
Other committee members also pressed for clearer targets in evaluating the goals for COVID vaccines, and for more robust data.
Like his fellow VRBPAC members, Cody Meissner, MD, of Dartmouth’s Geisel School of Medicine, Hanover, N.H., supported a move toward harmonizing the strains used in different companies’ vaccines. But he added that it wasn’t clear yet how frequently they should be administered.
“We need to see what happens with disease burden,” Dr. Meissner said. “We may or may not need annual vaccination. It’s just awfully early, it seems to me, in this process to answer that question.”
Among those serving on VRBPAC was one of the FDA’s more vocal critics on these points, Paul A. Offit, MD, a vaccine expert from Children’s Hospital of Philadelphia. Dr. Offit, for example, joined former FDA officials in writing a November opinion article for the Washington Post, arguing that the evidence for boosters for healthy younger adults was not strong.
At the Jan. 26 meeting, he supported the drive toward simplification of COVID vaccine schedules, while arguing for more data about how well these products are working.
“This virus is going to be with us for years, if not decades, and there will always be vulnerable groups who are going to be hospitalized and killed by the virus,” Dr. Offit said.
The CDC needs to provide more information about the characteristics of people being hospitalized with COVID infections, including their ages and comorbidities as well as details about their vaccine history, he said. In addition, academic researchers should provide a clearer picture of what immunological predictors are at play in increasing people’s risk from COVID.
“Then and only then can we really best make the decision about who gets vaccinated with what and when,” Dr. Offit said.
VRBPAC member Ofer Levy, MD, PhD, also urged the FDA to press for a collection of more robust and detailed information about the immune response to COVID-19 vaccinations, such as a deeper look at what’s happening with antibodies.
“I hope FDA will continue to reflect on how to best take this information forward, and encourage – or require – sponsors to gather more information in a standardized way across these different arms of the human immune system,” Dr. Levy said. “So we keep learning and keep doing this better.”
In recapping the panel’s suggestions at the end of the meeting, Peter Marks, MD, PhD, the director of the FDA’s Center for Biologics Evaluation and Research, addressed the requests made during the day’s meeting about better data on how the vaccines work.
“We heard loud and clear that we need to use a data-driven approach to get to the simplest possible scheme that we can for vaccination,” Dr. Marks said. “And it should be as simple as possible but not oversimplified, a little bit like they say about Mozart’s music.”
A version of this article first appeared on WebMD.com.
FDA okays Tidepool Loop app to help guide insulin delivery
The Food and Drug Administration has cleared the Tidepool Loop, a mobile application for use with compatible continuous glucose monitors (CGMs) and insulin pumps to enable automated insulin delivery.
Indicated for people with type 1 diabetes ages 6 years and up, the app algorithm was developed by the diabetes startup Tidepool, which already hosts a cloud-based platform for users to download and review data from different glucose meters, insulin pumps, and CGM systems. The Tidepool Loop project arose from patient-led, open-source initiatives to enable interoperability between the devices.
“The [FDA] authorization of the Tidepool Loop is a huge win for the type 1 diabetes (T1D) community and is a vital step towards a world where people with T1D can choose the pump, CGM, and algorithm that are best for them – and have all three work together seamlessly,” Aaron Kowalski, PhD, CEO of the advocacy organization JDRF, said in a statement.
JDRF helped support preclinical and clinical research in the development of the Loop algorithm, along with The Leona M. and Harry B. Helmsley Charitable Trust, the Tullman Foundation, and partnerships with device makers and donations from the T1D community.
Available by prescription only, the Tidepool app is for single patient use. It works with designated “integrated CGMs” and “alternate controller enabled pumps” to automatically increase, decrease, or suspend insulin delivery, based on the glucose readings and predicted values. The app can also recommend correction doses, which the user can confirm.
According to an FDA statement:“Tidepool Loop’s algorithm technology is designed to be compatible with other individual interoperable devices that meet prespecified acceptance criteria set forth in a validation and integration plan provided by the sponsor and cleared by the FDA as part of the premarket submission.”
Tidepool is finalizing agreements with the various device manufacturers “to create a seamless experience for both physicians prescribing Tidepool Loop and the patients using it,” according to a company statement.
Tidepool’s initial launch device partners have not yet been announced, but the company “has a development partnership with Dexcom and other yet-to-be-named medical device companies for future inclusion of their components with the Tidepool Loop platform,” the statement says.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration has cleared the Tidepool Loop, a mobile application for use with compatible continuous glucose monitors (CGMs) and insulin pumps to enable automated insulin delivery.
Indicated for people with type 1 diabetes ages 6 years and up, the app algorithm was developed by the diabetes startup Tidepool, which already hosts a cloud-based platform for users to download and review data from different glucose meters, insulin pumps, and CGM systems. The Tidepool Loop project arose from patient-led, open-source initiatives to enable interoperability between the devices.
“The [FDA] authorization of the Tidepool Loop is a huge win for the type 1 diabetes (T1D) community and is a vital step towards a world where people with T1D can choose the pump, CGM, and algorithm that are best for them – and have all three work together seamlessly,” Aaron Kowalski, PhD, CEO of the advocacy organization JDRF, said in a statement.
JDRF helped support preclinical and clinical research in the development of the Loop algorithm, along with The Leona M. and Harry B. Helmsley Charitable Trust, the Tullman Foundation, and partnerships with device makers and donations from the T1D community.
Available by prescription only, the Tidepool app is for single patient use. It works with designated “integrated CGMs” and “alternate controller enabled pumps” to automatically increase, decrease, or suspend insulin delivery, based on the glucose readings and predicted values. The app can also recommend correction doses, which the user can confirm.
According to an FDA statement:“Tidepool Loop’s algorithm technology is designed to be compatible with other individual interoperable devices that meet prespecified acceptance criteria set forth in a validation and integration plan provided by the sponsor and cleared by the FDA as part of the premarket submission.”
Tidepool is finalizing agreements with the various device manufacturers “to create a seamless experience for both physicians prescribing Tidepool Loop and the patients using it,” according to a company statement.
Tidepool’s initial launch device partners have not yet been announced, but the company “has a development partnership with Dexcom and other yet-to-be-named medical device companies for future inclusion of their components with the Tidepool Loop platform,” the statement says.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration has cleared the Tidepool Loop, a mobile application for use with compatible continuous glucose monitors (CGMs) and insulin pumps to enable automated insulin delivery.
Indicated for people with type 1 diabetes ages 6 years and up, the app algorithm was developed by the diabetes startup Tidepool, which already hosts a cloud-based platform for users to download and review data from different glucose meters, insulin pumps, and CGM systems. The Tidepool Loop project arose from patient-led, open-source initiatives to enable interoperability between the devices.
“The [FDA] authorization of the Tidepool Loop is a huge win for the type 1 diabetes (T1D) community and is a vital step towards a world where people with T1D can choose the pump, CGM, and algorithm that are best for them – and have all three work together seamlessly,” Aaron Kowalski, PhD, CEO of the advocacy organization JDRF, said in a statement.
JDRF helped support preclinical and clinical research in the development of the Loop algorithm, along with The Leona M. and Harry B. Helmsley Charitable Trust, the Tullman Foundation, and partnerships with device makers and donations from the T1D community.
Available by prescription only, the Tidepool app is for single patient use. It works with designated “integrated CGMs” and “alternate controller enabled pumps” to automatically increase, decrease, or suspend insulin delivery, based on the glucose readings and predicted values. The app can also recommend correction doses, which the user can confirm.
According to an FDA statement:“Tidepool Loop’s algorithm technology is designed to be compatible with other individual interoperable devices that meet prespecified acceptance criteria set forth in a validation and integration plan provided by the sponsor and cleared by the FDA as part of the premarket submission.”
Tidepool is finalizing agreements with the various device manufacturers “to create a seamless experience for both physicians prescribing Tidepool Loop and the patients using it,” according to a company statement.
Tidepool’s initial launch device partners have not yet been announced, but the company “has a development partnership with Dexcom and other yet-to-be-named medical device companies for future inclusion of their components with the Tidepool Loop platform,” the statement says.
A version of this article first appeared on Medscape.com.
FDA wants annual COVID boosters, just like annual flu shots
The U.S. Food and Drug Administration is suggesting a single annual shot. The formulation would be selected in June targeting the most threatening COVID-19 strains, and then people could get a shot in the fall when people begin spending more time indoors and exposure increases.
Some people, such as those who are older or immunocompromised, may need more than one dose.
A national advisory committee is expected to vote on the proposal at a meeting Jan. 26.
People in the United States have been much less likely to get an updated COVID-19 booster shot, compared with widespread uptake of the primary vaccine series. In its proposal, the FDA indicated it hoped a single annual shot would overcome challenges created by the complexity of the process – both in messaging and administration – attributed to that low booster rate. Nine in 10 people age 12 or older got the primary vaccine series in the United States, but only 15% got the latest booster shot for COVID-19.
About half of children and adults in the U.S. get an annual flu shot, according to Centers for Disease Control and Prevention data.
The FDA also wants to move to a single COVID-19 vaccine formulation that would be used for primary vaccine series and for booster shots.
COVID-19 cases, hospitalizations, and deaths are trending downward, according to the data tracker from the New York Times. Cases are down 28%, with 47,290 tallied daily. Hospitalizations are down 22%, with 37,474 daily. Deaths are down 4%, with an average of 489 per day as of Jan. 22.
A version of this article originally appeared on WebMD.com.
The U.S. Food and Drug Administration is suggesting a single annual shot. The formulation would be selected in June targeting the most threatening COVID-19 strains, and then people could get a shot in the fall when people begin spending more time indoors and exposure increases.
Some people, such as those who are older or immunocompromised, may need more than one dose.
A national advisory committee is expected to vote on the proposal at a meeting Jan. 26.
People in the United States have been much less likely to get an updated COVID-19 booster shot, compared with widespread uptake of the primary vaccine series. In its proposal, the FDA indicated it hoped a single annual shot would overcome challenges created by the complexity of the process – both in messaging and administration – attributed to that low booster rate. Nine in 10 people age 12 or older got the primary vaccine series in the United States, but only 15% got the latest booster shot for COVID-19.
About half of children and adults in the U.S. get an annual flu shot, according to Centers for Disease Control and Prevention data.
The FDA also wants to move to a single COVID-19 vaccine formulation that would be used for primary vaccine series and for booster shots.
COVID-19 cases, hospitalizations, and deaths are trending downward, according to the data tracker from the New York Times. Cases are down 28%, with 47,290 tallied daily. Hospitalizations are down 22%, with 37,474 daily. Deaths are down 4%, with an average of 489 per day as of Jan. 22.
A version of this article originally appeared on WebMD.com.
The U.S. Food and Drug Administration is suggesting a single annual shot. The formulation would be selected in June targeting the most threatening COVID-19 strains, and then people could get a shot in the fall when people begin spending more time indoors and exposure increases.
Some people, such as those who are older or immunocompromised, may need more than one dose.
A national advisory committee is expected to vote on the proposal at a meeting Jan. 26.
People in the United States have been much less likely to get an updated COVID-19 booster shot, compared with widespread uptake of the primary vaccine series. In its proposal, the FDA indicated it hoped a single annual shot would overcome challenges created by the complexity of the process – both in messaging and administration – attributed to that low booster rate. Nine in 10 people age 12 or older got the primary vaccine series in the United States, but only 15% got the latest booster shot for COVID-19.
About half of children and adults in the U.S. get an annual flu shot, according to Centers for Disease Control and Prevention data.
The FDA also wants to move to a single COVID-19 vaccine formulation that would be used for primary vaccine series and for booster shots.
COVID-19 cases, hospitalizations, and deaths are trending downward, according to the data tracker from the New York Times. Cases are down 28%, with 47,290 tallied daily. Hospitalizations are down 22%, with 37,474 daily. Deaths are down 4%, with an average of 489 per day as of Jan. 22.
A version of this article originally appeared on WebMD.com.