Christopher Palmer

The Food and Drug Administration has cleared the way for marketing of the BrainsWay deep transcranial magnetic stimulation system for treating patients with treatment-resistant obsessive-compulsive disorder (OCD).

“With today’s marketing authorization, patients with OCD who have not responded to traditional treatments now have another option,” said Carlos Peña, PhD, director of the division of neurological and physical medicine devices in the FDA’s Center for Devices and Radiological Health, in an Aug. 17 news release.

The clearance is based on results of a randomized, multicenter study of 100 patients,49 of whom were treated with the BrainsWay device and 51 of whom with a sham device. Of the patients treated with the BrainsWay device, 38% had a 30% or greater reduction in their Yale-Brown Obsessive Compulsive Scale scores, which measures severity of OCD symptoms, whereas only 11% treated with the sham device experienced such a reduction.

The most common adverse reaction was headache, experienced by 37.5% of patients treated with the BrainsWay device and 35.0% of those treated with the sham device. No serious adverse events were reported. Other common reactions included application site pain or discomfort, spasm or twitching, and jaw, facial, muscle, or neck pain; all of those were reported as mild or moderate and resolved quickly after each treatment was completed.

The device is contraindicated in patients with any sort of metal in or near their heads, such as cochlear implants or vagus nerve stimulators. Patients with a history of seizure should discuss it with their health care clinician.

Transcranial magnetic stimulation also has been approved to treat major depressive disorder and migraine with aura.

The full press release can be found on the FDA website.

[email protected]

The Food and Drug Administration has cleared the way for marketing of the BrainsWay deep transcranial magnetic stimulation system for treating patients with treatment-resistant obsessive-compulsive disorder (OCD).

“With today’s marketing authorization, patients with OCD who have not responded to traditional treatments now have another option,” said Carlos Peña, PhD, director of the division of neurological and physical medicine devices in the FDA’s Center for Devices and Radiological Health, in an Aug. 17 news release.

The clearance is based on results of a randomized, multicenter study of 100 patients,49 of whom were treated with the BrainsWay device and 51 of whom with a sham device. Of the patients treated with the BrainsWay device, 38% had a 30% or greater reduction in their Yale-Brown Obsessive Compulsive Scale scores, which measures severity of OCD symptoms, whereas only 11% treated with the sham device experienced such a reduction.

The most common adverse reaction was headache, experienced by 37.5% of patients treated with the BrainsWay device and 35.0% of those treated with the sham device. No serious adverse events were reported. Other common reactions included application site pain or discomfort, spasm or twitching, and jaw, facial, muscle, or neck pain; all of those were reported as mild or moderate and resolved quickly after each treatment was completed.

The device is contraindicated in patients with any sort of metal in or near their heads, such as cochlear implants or vagus nerve stimulators. Patients with a history of seizure should discuss it with their health care clinician.

Transcranial magnetic stimulation also has been approved to treat major depressive disorder and migraine with aura.

The full press release can be found on the FDA website.

[email protected]

The Food and Drug Administration has cleared the way for marketing of the BrainsWay deep transcranial magnetic stimulation system for treating patients with treatment-resistant obsessive-compulsive disorder (OCD).

“With today’s marketing authorization, patients with OCD who have not responded to traditional treatments now have another option,” said Carlos Peña, PhD, director of the division of neurological and physical medicine devices in the FDA’s Center for Devices and Radiological Health, in an Aug. 17 news release.

The clearance is based on results of a randomized, multicenter study of 100 patients,49 of whom were treated with the BrainsWay device and 51 of whom with a sham device. Of the patients treated with the BrainsWay device, 38% had a 30% or greater reduction in their Yale-Brown Obsessive Compulsive Scale scores, which measures severity of OCD symptoms, whereas only 11% treated with the sham device experienced such a reduction.

The most common adverse reaction was headache, experienced by 37.5% of patients treated with the BrainsWay device and 35.0% of those treated with the sham device. No serious adverse events were reported. Other common reactions included application site pain or discomfort, spasm or twitching, and jaw, facial, muscle, or neck pain; all of those were reported as mild or moderate and resolved quickly after each treatment was completed.

The device is contraindicated in patients with any sort of metal in or near their heads, such as cochlear implants or vagus nerve stimulators. Patients with a history of seizure should discuss it with their health care clinician.

Transcranial magnetic stimulation also has been approved to treat major depressive disorder and migraine with aura.

The full press release can be found on the FDA website.

[email protected]

Postapproval results for SynCardia Systems’ Companion 2 (C2) driver system for temporary total artificial hearts (TAH-t) have shown higher mortality and stroke rates than were seen with the previous system, the circulatory support system. As a result, the Food and Drug Administration has issued a safety alert cautioning them to weigh the risks and benefits carefully. The alert, issued on August 17, is based on a postapproval study conducted by SynCardia Systems.

Furthermore, patients and health care professionals are encouraged to report any adverse events using the FDA’s Medwatch reporting form, as well as return any devices associated with adverse events to the SynCardia Systems to help them and the FDA better understand the issue.

The C2 driver system is an external pneumatic system that activates an implanted TAH-t in eligible heart failure patients who have severe biventricular failure and are waiting for transplant. It is smaller than its predecessor, but per the device’s approved use, patients must still remain in the hospital while on the device. Since its approval in 2012, the Freedom driver system was approved in 2014, which allows patients to return home.

The full safety alert can be found on the FDA website.
 

[email protected]

Postapproval results for SynCardia Systems’ Companion 2 (C2) driver system for temporary total artificial hearts (TAH-t) have shown higher mortality and stroke rates than were seen with the previous system, the circulatory support system. As a result, the Food and Drug Administration has issued a safety alert cautioning them to weigh the risks and benefits carefully. The alert, issued on August 17, is based on a postapproval study conducted by SynCardia Systems.

Furthermore, patients and health care professionals are encouraged to report any adverse events using the FDA’s Medwatch reporting form, as well as return any devices associated with adverse events to the SynCardia Systems to help them and the FDA better understand the issue.

The C2 driver system is an external pneumatic system that activates an implanted TAH-t in eligible heart failure patients who have severe biventricular failure and are waiting for transplant. It is smaller than its predecessor, but per the device’s approved use, patients must still remain in the hospital while on the device. Since its approval in 2012, the Freedom driver system was approved in 2014, which allows patients to return home.

The full safety alert can be found on the FDA website.
 

[email protected]

Postapproval results for SynCardia Systems’ Companion 2 (C2) driver system for temporary total artificial hearts (TAH-t) have shown higher mortality and stroke rates than were seen with the previous system, the circulatory support system. As a result, the Food and Drug Administration has issued a safety alert cautioning them to weigh the risks and benefits carefully. The alert, issued on August 17, is based on a postapproval study conducted by SynCardia Systems.

Furthermore, patients and health care professionals are encouraged to report any adverse events using the FDA’s Medwatch reporting form, as well as return any devices associated with adverse events to the SynCardia Systems to help them and the FDA better understand the issue.

The C2 driver system is an external pneumatic system that activates an implanted TAH-t in eligible heart failure patients who have severe biventricular failure and are waiting for transplant. It is smaller than its predecessor, but per the device’s approved use, patients must still remain in the hospital while on the device. Since its approval in 2012, the Freedom driver system was approved in 2014, which allows patients to return home.

The full safety alert can be found on the FDA website.
 

[email protected]

The Food and Drug Administration has approved the first generic EpiPen and EpiPen Jr. autoinjector for the emergency treatment of allergic reactions, including anaphylaxis, for adults and children weighing more than 33 pounds, according to an announcement from the agency.

“Today’s approval of the first generic version of the most widely prescribed epinephrine autoinjector in the U.S. is part of our longstanding commitment to advance access to lower cost, safe, and effective generic alternatives once patents and other exclusivities no longer prevent approval,” FDA commissioner Scott Gottlieb, MD, said in the release.

Manufactured by Teva Pharmaceuticals USA, the two strengths of the generic versions are 0.3 mg and 0.15 mg.

The FDA has previously approved other epinephrine autoinjectors, which include brand-name products and so-called “authorized generic” versions of Epi-Pen and Adrenaclick. An authorized generic “is made under the brand name’s existing drug application using the same formulation, process, and manufacturing facilities that are used by the brand name manufacturer. The labeling or packaging is, however, changed to remove the brand name or other trade dress. In some cases, a company may choose to sell an authorized generic at a lower cost than the brand-name drug product,” according to the FDA statement.

“Complex” generics – those that, as with this generic, include both a drug and a delivery device – face a tougher path to approval because the FDA has to evaluate and approve both components. “We remain committed to doing our part to provide scientific and regulatory clarity for sponsors seeking to develop complex generics, as well as prioritize the approval of medicines with little or no generic competition, as part of our overarching effort to remove barriers to generic development and market entry of critically important medicines,” Dr. Gottlieb explained.

Side effects of epinephrine autoinjectors include anxiety, restlessness, palpitations, nausea, and weakness; rarely, serious skin and soft-tissue infections after use of epinephrine autoinjectors have been reported.

Find more information about this approval in the FDA press announcement.

[email protected]

The Food and Drug Administration has approved the first generic EpiPen and EpiPen Jr. autoinjector for the emergency treatment of allergic reactions, including anaphylaxis, for adults and children weighing more than 33 pounds, according to an announcement from the agency.

“Today’s approval of the first generic version of the most widely prescribed epinephrine autoinjector in the U.S. is part of our longstanding commitment to advance access to lower cost, safe, and effective generic alternatives once patents and other exclusivities no longer prevent approval,” FDA commissioner Scott Gottlieb, MD, said in the release.

Manufactured by Teva Pharmaceuticals USA, the two strengths of the generic versions are 0.3 mg and 0.15 mg.

The FDA has previously approved other epinephrine autoinjectors, which include brand-name products and so-called “authorized generic” versions of Epi-Pen and Adrenaclick. An authorized generic “is made under the brand name’s existing drug application using the same formulation, process, and manufacturing facilities that are used by the brand name manufacturer. The labeling or packaging is, however, changed to remove the brand name or other trade dress. In some cases, a company may choose to sell an authorized generic at a lower cost than the brand-name drug product,” according to the FDA statement.

“Complex” generics – those that, as with this generic, include both a drug and a delivery device – face a tougher path to approval because the FDA has to evaluate and approve both components. “We remain committed to doing our part to provide scientific and regulatory clarity for sponsors seeking to develop complex generics, as well as prioritize the approval of medicines with little or no generic competition, as part of our overarching effort to remove barriers to generic development and market entry of critically important medicines,” Dr. Gottlieb explained.

Side effects of epinephrine autoinjectors include anxiety, restlessness, palpitations, nausea, and weakness; rarely, serious skin and soft-tissue infections after use of epinephrine autoinjectors have been reported.

Find more information about this approval in the FDA press announcement.

[email protected]

The Food and Drug Administration has approved the first generic EpiPen and EpiPen Jr. autoinjector for the emergency treatment of allergic reactions, including anaphylaxis, for adults and children weighing more than 33 pounds, according to an announcement from the agency.

“Today’s approval of the first generic version of the most widely prescribed epinephrine autoinjector in the U.S. is part of our longstanding commitment to advance access to lower cost, safe, and effective generic alternatives once patents and other exclusivities no longer prevent approval,” FDA commissioner Scott Gottlieb, MD, said in the release.

Manufactured by Teva Pharmaceuticals USA, the two strengths of the generic versions are 0.3 mg and 0.15 mg.

The FDA has previously approved other epinephrine autoinjectors, which include brand-name products and so-called “authorized generic” versions of Epi-Pen and Adrenaclick. An authorized generic “is made under the brand name’s existing drug application using the same formulation, process, and manufacturing facilities that are used by the brand name manufacturer. The labeling or packaging is, however, changed to remove the brand name or other trade dress. In some cases, a company may choose to sell an authorized generic at a lower cost than the brand-name drug product,” according to the FDA statement.

“Complex” generics – those that, as with this generic, include both a drug and a delivery device – face a tougher path to approval because the FDA has to evaluate and approve both components. “We remain committed to doing our part to provide scientific and regulatory clarity for sponsors seeking to develop complex generics, as well as prioritize the approval of medicines with little or no generic competition, as part of our overarching effort to remove barriers to generic development and market entry of critically important medicines,” Dr. Gottlieb explained.

Side effects of epinephrine autoinjectors include anxiety, restlessness, palpitations, nausea, and weakness; rarely, serious skin and soft-tissue infections after use of epinephrine autoinjectors have been reported.

Find more information about this approval in the FDA press announcement.

[email protected]

The Food and Drug Administration has approved Kalydeco (ivacaftor) for the treatment of patients aged 12 to less than 24 months who have cystic fibrosis that is caused by any of 10 mutations in the CFTR gene and is responsive to the drug, the drug’s developer announced.

The drug was approved for patients aged 6 years and older in 2012 and in patients aged 2-5 years in 2015 and is the only approved drug that treats the underlying cause of cystic fibrosis rather than its symptoms.

The approval is based on the ongoing phase 3, open-label ARRIVAL trial (NCT02725567), which is assessing the drug’s safety in children aged 12 months to less than 24 months. The trial’s investigators have found that its safety profile in this age group is consistent with that seen in older children and adults. Most adverse events were mild to moderate; the most common (occurring in more than 30% of patients) were cough, pyrexia, elevated aspartate aminotransferase, elevated alanine aminotransferase, and runny nose. The trial found that, after 24 weeks of treatment, the mean sweat chloride levels decreased from 104.1 mmol/L (n = 14) to 33.8 mmol/L (n = 14).

Ivacaftor is contraindicated in patients taking certain antibiotics, seizure medications, or other medications; risk of drug interaction – affecting either the performance of ivacaftor or that of the other medication – is also a concern. Patients should inform their doctors if they are pregnant, planning to become pregnant, or breastfeeding; have liver or kidney problems; or drink grapefruit juice or eat grapefruit or Seville oranges. There also is a risk of high liver enzymes or cataracts. Ivacaftor is available in 150-mg tablets for adults and pediatric patients aged 6 years and older and in 50-mg and 75-mg granules for younger patients. Full prescribing information can be found on the FDA website.

[email protected]
 

The Food and Drug Administration has approved Kalydeco (ivacaftor) for the treatment of patients aged 12 to less than 24 months who have cystic fibrosis that is caused by any of 10 mutations in the CFTR gene and is responsive to the drug, the drug’s developer announced.

The drug was approved for patients aged 6 years and older in 2012 and in patients aged 2-5 years in 2015 and is the only approved drug that treats the underlying cause of cystic fibrosis rather than its symptoms.

The approval is based on the ongoing phase 3, open-label ARRIVAL trial (NCT02725567), which is assessing the drug’s safety in children aged 12 months to less than 24 months. The trial’s investigators have found that its safety profile in this age group is consistent with that seen in older children and adults. Most adverse events were mild to moderate; the most common (occurring in more than 30% of patients) were cough, pyrexia, elevated aspartate aminotransferase, elevated alanine aminotransferase, and runny nose. The trial found that, after 24 weeks of treatment, the mean sweat chloride levels decreased from 104.1 mmol/L (n = 14) to 33.8 mmol/L (n = 14).

Ivacaftor is contraindicated in patients taking certain antibiotics, seizure medications, or other medications; risk of drug interaction – affecting either the performance of ivacaftor or that of the other medication – is also a concern. Patients should inform their doctors if they are pregnant, planning to become pregnant, or breastfeeding; have liver or kidney problems; or drink grapefruit juice or eat grapefruit or Seville oranges. There also is a risk of high liver enzymes or cataracts. Ivacaftor is available in 150-mg tablets for adults and pediatric patients aged 6 years and older and in 50-mg and 75-mg granules for younger patients. Full prescribing information can be found on the FDA website.

[email protected]
 

The Food and Drug Administration has approved Kalydeco (ivacaftor) for the treatment of patients aged 12 to less than 24 months who have cystic fibrosis that is caused by any of 10 mutations in the CFTR gene and is responsive to the drug, the drug’s developer announced.

The drug was approved for patients aged 6 years and older in 2012 and in patients aged 2-5 years in 2015 and is the only approved drug that treats the underlying cause of cystic fibrosis rather than its symptoms.

The approval is based on the ongoing phase 3, open-label ARRIVAL trial (NCT02725567), which is assessing the drug’s safety in children aged 12 months to less than 24 months. The trial’s investigators have found that its safety profile in this age group is consistent with that seen in older children and adults. Most adverse events were mild to moderate; the most common (occurring in more than 30% of patients) were cough, pyrexia, elevated aspartate aminotransferase, elevated alanine aminotransferase, and runny nose. The trial found that, after 24 weeks of treatment, the mean sweat chloride levels decreased from 104.1 mmol/L (n = 14) to 33.8 mmol/L (n = 14).

Ivacaftor is contraindicated in patients taking certain antibiotics, seizure medications, or other medications; risk of drug interaction – affecting either the performance of ivacaftor or that of the other medication – is also a concern. Patients should inform their doctors if they are pregnant, planning to become pregnant, or breastfeeding; have liver or kidney problems; or drink grapefruit juice or eat grapefruit or Seville oranges. There also is a risk of high liver enzymes or cataracts. Ivacaftor is available in 150-mg tablets for adults and pediatric patients aged 6 years and older and in 50-mg and 75-mg granules for younger patients. Full prescribing information can be found on the FDA website.

[email protected]
 

In combination with an optimized background regimen, the humanized IgG4 monoclonal antibody ibalizumab can reduce viral load and increase CD4 counts in patients with multidrug-resistant HIV type 1 (MDR HIV-1), according to a study published in the New England Journal of Medicine. It does so by binding noncompetitively to CD4 receptors, which can thereby block HIV-1 entry.

copyright alexskopje/Thinkstock

TMB-301 (NCT02475629) was a 24-week, open-label, single-group, phase 3 study that included patients with MDR HIV-1. During July 2015 to October 2016, patients first continued on their previous treatment for 7 days, then were given a 2,000-mg loading dose of ibalizumab as monotherapy, and then continued on 800 mg ibalizumab every 14 days plus an individualized regimen that was optimized based on patients’ past treatments for the remainder of the study period.

The primary endpoint was how many patients experienced a decrease in viral load of at least 0.5 log₁₀ copies/mL from baseline to day 14 – that is, after the 7-day control period plus the 7-days ibalizumab monotherapy period. This occurred in 33 of the 40 patients (83%; 95% confidence interval, 67%-93%; P less than .001); in fact, 60% of patients (n = 24) experienced a decrease of at least 1.0 log₁₀ copies/mL during that period, and the mean and median reductions were 1.1 log₁₀ copies/mL. One secondary end point was increase in CD4 counts: The mean increase was 62 cells/mcL, although smaller increases were seen among patients with lower starting counts. Of the 10 patients who exhibited virologic failure or rebound, 9 showed less susceptibility to ibalizumab at week 25 than they had shown at baseline.

At least one adverse event was seen in 32 patients (80%), although most (87%) of those events were mild to moderate in severity, such as diarrhea (20%) and nausea, fatigue, pyrexia, rash, and dizziness (13% each). More serious events (grade 3 or 4) were seen in 28% of patients (n = 11); serious events were seen in nine patients – including immune reconstitution inflammatory syndrome in one. Four patients died during the study, although none of the deaths were believed to have been related to treatment with ibalizumab.

The investigators noted that the study’s limitations – its small size, uncontrolled design, and limited time for analyzing secondary and safety endpoints – were largely related to small number of patients with MDR HIV-1. The investigators noted that, in accordance with HIV treatment guidelines, patients who experience resistance-related treatment failure should be started on a regimen that includes at least two fully active agents; the investigators suggest that the addition of a drug like ibalizumab provides new opportunities for effective treatments in these situations.

This study was originally presented at the Conference on Retroviruses & Opportunistic Infections in partnership with the International Antiviral Society.

The study was supported by the Orphan Products Clinical Trials Grants Program of the Food and Drug Administration and TaiMed, which developed ibalizumab. The authors disclosed ties to various industry entities, including grants, personal fees, nonfinancial support, and other forms of support from TaiMed.

[email protected]

SOURCE: Emu B et al. New Engl J Med. 2018 Aug 16;379(9):645-54.

In combination with an optimized background regimen, the humanized IgG4 monoclonal antibody ibalizumab can reduce viral load and increase CD4 counts in patients with multidrug-resistant HIV type 1 (MDR HIV-1), according to a study published in the New England Journal of Medicine. It does so by binding noncompetitively to CD4 receptors, which can thereby block HIV-1 entry.

copyright alexskopje/Thinkstock

TMB-301 (NCT02475629) was a 24-week, open-label, single-group, phase 3 study that included patients with MDR HIV-1. During July 2015 to October 2016, patients first continued on their previous treatment for 7 days, then were given a 2,000-mg loading dose of ibalizumab as monotherapy, and then continued on 800 mg ibalizumab every 14 days plus an individualized regimen that was optimized based on patients’ past treatments for the remainder of the study period.

The primary endpoint was how many patients experienced a decrease in viral load of at least 0.5 log₁₀ copies/mL from baseline to day 14 – that is, after the 7-day control period plus the 7-days ibalizumab monotherapy period. This occurred in 33 of the 40 patients (83%; 95% confidence interval, 67%-93%; P less than .001); in fact, 60% of patients (n = 24) experienced a decrease of at least 1.0 log₁₀ copies/mL during that period, and the mean and median reductions were 1.1 log₁₀ copies/mL. One secondary end point was increase in CD4 counts: The mean increase was 62 cells/mcL, although smaller increases were seen among patients with lower starting counts. Of the 10 patients who exhibited virologic failure or rebound, 9 showed less susceptibility to ibalizumab at week 25 than they had shown at baseline.

At least one adverse event was seen in 32 patients (80%), although most (87%) of those events were mild to moderate in severity, such as diarrhea (20%) and nausea, fatigue, pyrexia, rash, and dizziness (13% each). More serious events (grade 3 or 4) were seen in 28% of patients (n = 11); serious events were seen in nine patients – including immune reconstitution inflammatory syndrome in one. Four patients died during the study, although none of the deaths were believed to have been related to treatment with ibalizumab.

The investigators noted that the study’s limitations – its small size, uncontrolled design, and limited time for analyzing secondary and safety endpoints – were largely related to small number of patients with MDR HIV-1. The investigators noted that, in accordance with HIV treatment guidelines, patients who experience resistance-related treatment failure should be started on a regimen that includes at least two fully active agents; the investigators suggest that the addition of a drug like ibalizumab provides new opportunities for effective treatments in these situations.

This study was originally presented at the Conference on Retroviruses & Opportunistic Infections in partnership with the International Antiviral Society.

The study was supported by the Orphan Products Clinical Trials Grants Program of the Food and Drug Administration and TaiMed, which developed ibalizumab. The authors disclosed ties to various industry entities, including grants, personal fees, nonfinancial support, and other forms of support from TaiMed.

[email protected]

SOURCE: Emu B et al. New Engl J Med. 2018 Aug 16;379(9):645-54.

In combination with an optimized background regimen, the humanized IgG4 monoclonal antibody ibalizumab can reduce viral load and increase CD4 counts in patients with multidrug-resistant HIV type 1 (MDR HIV-1), according to a study published in the New England Journal of Medicine. It does so by binding noncompetitively to CD4 receptors, which can thereby block HIV-1 entry.

copyright alexskopje/Thinkstock

TMB-301 (NCT02475629) was a 24-week, open-label, single-group, phase 3 study that included patients with MDR HIV-1. During July 2015 to October 2016, patients first continued on their previous treatment for 7 days, then were given a 2,000-mg loading dose of ibalizumab as monotherapy, and then continued on 800 mg ibalizumab every 14 days plus an individualized regimen that was optimized based on patients’ past treatments for the remainder of the study period.

The primary endpoint was how many patients experienced a decrease in viral load of at least 0.5 log₁₀ copies/mL from baseline to day 14 – that is, after the 7-day control period plus the 7-days ibalizumab monotherapy period. This occurred in 33 of the 40 patients (83%; 95% confidence interval, 67%-93%; P less than .001); in fact, 60% of patients (n = 24) experienced a decrease of at least 1.0 log₁₀ copies/mL during that period, and the mean and median reductions were 1.1 log₁₀ copies/mL. One secondary end point was increase in CD4 counts: The mean increase was 62 cells/mcL, although smaller increases were seen among patients with lower starting counts. Of the 10 patients who exhibited virologic failure or rebound, 9 showed less susceptibility to ibalizumab at week 25 than they had shown at baseline.

At least one adverse event was seen in 32 patients (80%), although most (87%) of those events were mild to moderate in severity, such as diarrhea (20%) and nausea, fatigue, pyrexia, rash, and dizziness (13% each). More serious events (grade 3 or 4) were seen in 28% of patients (n = 11); serious events were seen in nine patients – including immune reconstitution inflammatory syndrome in one. Four patients died during the study, although none of the deaths were believed to have been related to treatment with ibalizumab.

The investigators noted that the study’s limitations – its small size, uncontrolled design, and limited time for analyzing secondary and safety endpoints – were largely related to small number of patients with MDR HIV-1. The investigators noted that, in accordance with HIV treatment guidelines, patients who experience resistance-related treatment failure should be started on a regimen that includes at least two fully active agents; the investigators suggest that the addition of a drug like ibalizumab provides new opportunities for effective treatments in these situations.

This study was originally presented at the Conference on Retroviruses & Opportunistic Infections in partnership with the International Antiviral Society.

The study was supported by the Orphan Products Clinical Trials Grants Program of the Food and Drug Administration and TaiMed, which developed ibalizumab. The authors disclosed ties to various industry entities, including grants, personal fees, nonfinancial support, and other forms of support from TaiMed.

[email protected]

SOURCE: Emu B et al. New Engl J Med. 2018 Aug 16;379(9):645-54.

Neither heart rate nor blood pressure worsened under long-term use of long-acting beta₂ agonists olodaterol or formoterol in patients with chronic obstructive pulmonary disease (COPD), according to a post hoc pooled analysis published in Pulmonary Pharmacology & Therapeutics.

The study was conducted by Stefan Andreas, MD, department of cardiology and pneumology, University Medical Centre Göttingen, and Lung Clinic Immenhausen, Germany. The analysis evaluated data from four studies and included a total of 3,104 patients with moderate to very severe COPD, which was defined as Global Initiative for Chronic Obstructive Lung Disease stage 2-4. Patients were randomized to either once-daily olodaterol (5 or 10 mcg), twice-daily formoterol (12 mcg), or placebo. Heart rate and blood pressure were measured before and after dosing at baseline and at four time points during the study: 6 weeks, 12 weeks, 24 weeks, and 48 weeks.

At all time points, the increases seen in the placebo group were greater than seen in the treatment groups; both systolic and diastolic blood pressure showed either slight decreases from or similarities with those seen at baseline, depending on time point. Furthermore, short-term effects were seen around dosing, from before administration to after, although these changes were quantitatively small.

One limitation of the study is that it couldn’t include patients with unstable COPD because of safety reasons; this prevents the findings from being more broadly generalizable.

“These findings, in a large COPD database, speak against the potential negative cardiovascular effects of olodaterol, as well as those of formoterol,” the researchers concluded.

They reported personal fees from various industry entities, such as Novartis, AstraZeneca, and GlaxoSmithKline. Some also reported receiving personal fees from or working for Boehringer Ingelheim, which funded the work.

[email protected]

SOURCE: Andreas S et al. Pulm Pharmacol Ther. 2018 Aug 2. doi: 10.1016/j.pupt.2018.08.002.

Neither heart rate nor blood pressure worsened under long-term use of long-acting beta₂ agonists olodaterol or formoterol in patients with chronic obstructive pulmonary disease (COPD), according to a post hoc pooled analysis published in Pulmonary Pharmacology & Therapeutics.

The study was conducted by Stefan Andreas, MD, department of cardiology and pneumology, University Medical Centre Göttingen, and Lung Clinic Immenhausen, Germany. The analysis evaluated data from four studies and included a total of 3,104 patients with moderate to very severe COPD, which was defined as Global Initiative for Chronic Obstructive Lung Disease stage 2-4. Patients were randomized to either once-daily olodaterol (5 or 10 mcg), twice-daily formoterol (12 mcg), or placebo. Heart rate and blood pressure were measured before and after dosing at baseline and at four time points during the study: 6 weeks, 12 weeks, 24 weeks, and 48 weeks.

At all time points, the increases seen in the placebo group were greater than seen in the treatment groups; both systolic and diastolic blood pressure showed either slight decreases from or similarities with those seen at baseline, depending on time point. Furthermore, short-term effects were seen around dosing, from before administration to after, although these changes were quantitatively small.

One limitation of the study is that it couldn’t include patients with unstable COPD because of safety reasons; this prevents the findings from being more broadly generalizable.

“These findings, in a large COPD database, speak against the potential negative cardiovascular effects of olodaterol, as well as those of formoterol,” the researchers concluded.

They reported personal fees from various industry entities, such as Novartis, AstraZeneca, and GlaxoSmithKline. Some also reported receiving personal fees from or working for Boehringer Ingelheim, which funded the work.

[email protected]

SOURCE: Andreas S et al. Pulm Pharmacol Ther. 2018 Aug 2. doi: 10.1016/j.pupt.2018.08.002.

Neither heart rate nor blood pressure worsened under long-term use of long-acting beta₂ agonists olodaterol or formoterol in patients with chronic obstructive pulmonary disease (COPD), according to a post hoc pooled analysis published in Pulmonary Pharmacology & Therapeutics.

The study was conducted by Stefan Andreas, MD, department of cardiology and pneumology, University Medical Centre Göttingen, and Lung Clinic Immenhausen, Germany. The analysis evaluated data from four studies and included a total of 3,104 patients with moderate to very severe COPD, which was defined as Global Initiative for Chronic Obstructive Lung Disease stage 2-4. Patients were randomized to either once-daily olodaterol (5 or 10 mcg), twice-daily formoterol (12 mcg), or placebo. Heart rate and blood pressure were measured before and after dosing at baseline and at four time points during the study: 6 weeks, 12 weeks, 24 weeks, and 48 weeks.

At all time points, the increases seen in the placebo group were greater than seen in the treatment groups; both systolic and diastolic blood pressure showed either slight decreases from or similarities with those seen at baseline, depending on time point. Furthermore, short-term effects were seen around dosing, from before administration to after, although these changes were quantitatively small.

One limitation of the study is that it couldn’t include patients with unstable COPD because of safety reasons; this prevents the findings from being more broadly generalizable.

“These findings, in a large COPD database, speak against the potential negative cardiovascular effects of olodaterol, as well as those of formoterol,” the researchers concluded.

They reported personal fees from various industry entities, such as Novartis, AstraZeneca, and GlaxoSmithKline. Some also reported receiving personal fees from or working for Boehringer Ingelheim, which funded the work.

[email protected]

SOURCE: Andreas S et al. Pulm Pharmacol Ther. 2018 Aug 2. doi: 10.1016/j.pupt.2018.08.002.

Janssen has applied to the Food and Drug Administration and the European Medicines Agency to allow splitting of the first infusion of daratumumab (Darzalex) in multiple myeloma patients over 2 consecutive days.

Courtesy Janssen Biotech

The goal is to improve the treatment experience for patients and physicians, according to the announcement from Janssen.

The regulatory submissions are based on the global, multi-arm, phase 1b MMY1001 study (NCT01998971). The study evaluated daratumumab in combination with various other treatments in 240 patients with multiple myeloma. It found that both the safety profile and the pharmacokinetics concentrations seen with either single dosing or split dosing were similar.

Daratumumab is the first approved monoclonal antibody that targets CD38, which is expressed across multiple myeloma cells regardless of disease stage. Daratumumab is currently approved for treatment of multiple myeloma in both the United States and the European Union either as monotherapy or in conjunction with other treatments.

Daratumumab is known to sometimes cause severe/serious infusion reactions, such as anaphylactic reactions; interfere with serological testing; and cause neutropenia or thrombocytopenia.

[email protected]

Janssen has applied to the Food and Drug Administration and the European Medicines Agency to allow splitting of the first infusion of daratumumab (Darzalex) in multiple myeloma patients over 2 consecutive days.

Courtesy Janssen Biotech

The goal is to improve the treatment experience for patients and physicians, according to the announcement from Janssen.

The regulatory submissions are based on the global, multi-arm, phase 1b MMY1001 study (NCT01998971). The study evaluated daratumumab in combination with various other treatments in 240 patients with multiple myeloma. It found that both the safety profile and the pharmacokinetics concentrations seen with either single dosing or split dosing were similar.

Daratumumab is the first approved monoclonal antibody that targets CD38, which is expressed across multiple myeloma cells regardless of disease stage. Daratumumab is currently approved for treatment of multiple myeloma in both the United States and the European Union either as monotherapy or in conjunction with other treatments.

Daratumumab is known to sometimes cause severe/serious infusion reactions, such as anaphylactic reactions; interfere with serological testing; and cause neutropenia or thrombocytopenia.

[email protected]

Janssen has applied to the Food and Drug Administration and the European Medicines Agency to allow splitting of the first infusion of daratumumab (Darzalex) in multiple myeloma patients over 2 consecutive days.

Courtesy Janssen Biotech

The goal is to improve the treatment experience for patients and physicians, according to the announcement from Janssen.

The regulatory submissions are based on the global, multi-arm, phase 1b MMY1001 study (NCT01998971). The study evaluated daratumumab in combination with various other treatments in 240 patients with multiple myeloma. It found that both the safety profile and the pharmacokinetics concentrations seen with either single dosing or split dosing were similar.

Daratumumab is the first approved monoclonal antibody that targets CD38, which is expressed across multiple myeloma cells regardless of disease stage. Daratumumab is currently approved for treatment of multiple myeloma in both the United States and the European Union either as monotherapy or in conjunction with other treatments.

Daratumumab is known to sometimes cause severe/serious infusion reactions, such as anaphylactic reactions; interfere with serological testing; and cause neutropenia or thrombocytopenia.

[email protected]

Women who were obese and had diabetes before becoming pregnant were sixfold more likely to have children with psychiatric and neurodevelopmental disorders by age 11 years, as compared to women with normal body mass indexes (BMIs), based on results of a large, prospective, population-based, cohort study published in Pediatrics.

The risks to offspring whose mothers were obese and had pregestational diabetes mellitus (PGDM) were far greater than the risks seen when mothers had either condition alone or had gestational diabetes mellitus (GDM) in the study, reported Linghua Kong of the Karolinska Institute, Stockholm, and colleagues. The study is based on data from various national registries in Finland regarding 649,043 live births during 2004-2014 and data regarding psychiatric diagnoses from the Finnish Care Registers for Health Care.

Of the children in the cohort, 7.67% had mothers who were obese and 3.66% had mothers who were severely obese based on standard World Health Organization criteria; mothers had PGDM in 0.62% of the births and GDM in 15.7% of the births.

Overall, 5.4% of the children were diagnosed with a psychiatric disorder by age 11 years.

Compared with children born to mothers of normal weight (BMI less than 25 kg/m²), those born to mothers with severe maternal obesity alone (BMI greater than 35) had higher rates of developmental disorders or speech, language, motor, and scholastic skills (hazard ratio, 1.69; 95% confidence interval 1.54-1.86); ADHD and/or conduct disorder (HR, 1.88; 95% CI, 1.58-2.23); and psychosis and mood and anxiety disorders (HR, 1.67; 95% CI, 1.31-2.13). Increased risk of psychiatric disorders were only slightly statistically significant in the offspring of women with severe obesity and GDM.

The risks were significantly elevated, however, for children born to obese women who also had PGDM. The hazard ratio for autism spectrum disorder was 6.49 (95% CI, 3.08-13.69), and the HR for ADHD and/or conduct disorder was 6.03 (95% CI, 3.23-11.24). The risks were fourfold higher for mixed disorders of emotions and conduct, disorders of social function, and tics (HR, 4.29; 95% CI, 2.14-8.60).

Limitations of the study included basing results on shorter follow-up times for those born later in the study period, grouping of offspring’s disorder diagnoses, basing the definition of PGDM on insulin prescription, and using BMI measurements taken at only one time point during pregnancy.

The researchers were supported by the National Institute for Health and Welfare: Drugs and Pregnancy project, the Swedish Research Council, the regional agreement on medical training and clinical research between Stockholm County Council and Karolinska Institutet Stockholm County Council, the China Scholarship Council, and the Swedish Brain Foundation.

[email protected]

SOURCE: Kong L et al. Pediatrics. 2018 Sep;142(3):1-11.

Women who were obese and had diabetes before becoming pregnant were sixfold more likely to have children with psychiatric and neurodevelopmental disorders by age 11 years, as compared to women with normal body mass indexes (BMIs), based on results of a large, prospective, population-based, cohort study published in Pediatrics.

The risks to offspring whose mothers were obese and had pregestational diabetes mellitus (PGDM) were far greater than the risks seen when mothers had either condition alone or had gestational diabetes mellitus (GDM) in the study, reported Linghua Kong of the Karolinska Institute, Stockholm, and colleagues. The study is based on data from various national registries in Finland regarding 649,043 live births during 2004-2014 and data regarding psychiatric diagnoses from the Finnish Care Registers for Health Care.

Of the children in the cohort, 7.67% had mothers who were obese and 3.66% had mothers who were severely obese based on standard World Health Organization criteria; mothers had PGDM in 0.62% of the births and GDM in 15.7% of the births.

Overall, 5.4% of the children were diagnosed with a psychiatric disorder by age 11 years.

Compared with children born to mothers of normal weight (BMI less than 25 kg/m²), those born to mothers with severe maternal obesity alone (BMI greater than 35) had higher rates of developmental disorders or speech, language, motor, and scholastic skills (hazard ratio, 1.69; 95% confidence interval 1.54-1.86); ADHD and/or conduct disorder (HR, 1.88; 95% CI, 1.58-2.23); and psychosis and mood and anxiety disorders (HR, 1.67; 95% CI, 1.31-2.13). Increased risk of psychiatric disorders were only slightly statistically significant in the offspring of women with severe obesity and GDM.

The risks were significantly elevated, however, for children born to obese women who also had PGDM. The hazard ratio for autism spectrum disorder was 6.49 (95% CI, 3.08-13.69), and the HR for ADHD and/or conduct disorder was 6.03 (95% CI, 3.23-11.24). The risks were fourfold higher for mixed disorders of emotions and conduct, disorders of social function, and tics (HR, 4.29; 95% CI, 2.14-8.60).

Limitations of the study included basing results on shorter follow-up times for those born later in the study period, grouping of offspring’s disorder diagnoses, basing the definition of PGDM on insulin prescription, and using BMI measurements taken at only one time point during pregnancy.

The researchers were supported by the National Institute for Health and Welfare: Drugs and Pregnancy project, the Swedish Research Council, the regional agreement on medical training and clinical research between Stockholm County Council and Karolinska Institutet Stockholm County Council, the China Scholarship Council, and the Swedish Brain Foundation.

[email protected]

SOURCE: Kong L et al. Pediatrics. 2018 Sep;142(3):1-11.

Women who were obese and had diabetes before becoming pregnant were sixfold more likely to have children with psychiatric and neurodevelopmental disorders by age 11 years, as compared to women with normal body mass indexes (BMIs), based on results of a large, prospective, population-based, cohort study published in Pediatrics.

The risks to offspring whose mothers were obese and had pregestational diabetes mellitus (PGDM) were far greater than the risks seen when mothers had either condition alone or had gestational diabetes mellitus (GDM) in the study, reported Linghua Kong of the Karolinska Institute, Stockholm, and colleagues. The study is based on data from various national registries in Finland regarding 649,043 live births during 2004-2014 and data regarding psychiatric diagnoses from the Finnish Care Registers for Health Care.

Of the children in the cohort, 7.67% had mothers who were obese and 3.66% had mothers who were severely obese based on standard World Health Organization criteria; mothers had PGDM in 0.62% of the births and GDM in 15.7% of the births.

Overall, 5.4% of the children were diagnosed with a psychiatric disorder by age 11 years.

Compared with children born to mothers of normal weight (BMI less than 25 kg/m²), those born to mothers with severe maternal obesity alone (BMI greater than 35) had higher rates of developmental disorders or speech, language, motor, and scholastic skills (hazard ratio, 1.69; 95% confidence interval 1.54-1.86); ADHD and/or conduct disorder (HR, 1.88; 95% CI, 1.58-2.23); and psychosis and mood and anxiety disorders (HR, 1.67; 95% CI, 1.31-2.13). Increased risk of psychiatric disorders were only slightly statistically significant in the offspring of women with severe obesity and GDM.

The risks were significantly elevated, however, for children born to obese women who also had PGDM. The hazard ratio for autism spectrum disorder was 6.49 (95% CI, 3.08-13.69), and the HR for ADHD and/or conduct disorder was 6.03 (95% CI, 3.23-11.24). The risks were fourfold higher for mixed disorders of emotions and conduct, disorders of social function, and tics (HR, 4.29; 95% CI, 2.14-8.60).

Limitations of the study included basing results on shorter follow-up times for those born later in the study period, grouping of offspring’s disorder diagnoses, basing the definition of PGDM on insulin prescription, and using BMI measurements taken at only one time point during pregnancy.

The researchers were supported by the National Institute for Health and Welfare: Drugs and Pregnancy project, the Swedish Research Council, the regional agreement on medical training and clinical research between Stockholm County Council and Karolinska Institutet Stockholm County Council, the China Scholarship Council, and the Swedish Brain Foundation.

[email protected]

SOURCE: Kong L et al. Pediatrics. 2018 Sep;142(3):1-11.

The Food and Drug Administration has expanded the indication for Orkambi (lumacaftor/ivacaftor) to include patients who are aged as young as 2 years with cystic fibrosis (CF), according to its manufacturer, Vertex Pharmaceuticals. Specifically, the drug is meant to treat the most common underlying cause of CF – having two copies of the F508del-CFTR mutation – and is the first drug to treat it.

The approval is based on a phase 3, two-part, open-label, multicenter study that assessed various doses in patents aged 2-5 years. The study demonstrated safety and tolerability in that age group equivalent to that seen in older patients. The drug is expected to be available for this age group within 2-4 weeks of this approval.

Available as oral granules in two doses for weight-based dosing (either lumacaftor 100 mg/ivacaftor 125 mg or lumacaftor 150 mg/ivacaftor 188 mg), the compound targets the defective chloride channels responsible for CF; the two halves work together to increase the number of chloride channels on cell surfaces and also improve their function.

Orkambi should be prescribed only for patients with CF who have the dual F508del-CFTR mutation; it is not indicated for other types of CF. Patients should not take this drug if they are taking drugs such as rifampin, phenytoin, triazolam, or cyclosporine because of possible drug interactions. It can also lead to worsening liver function and elevated blood liver enzymes, increased blood pressure, or cataracts. The most common side effects include breathing problems, nausea, fatigue, and rash. Full prescribing information is available on the FDA website.
 

[email protected]

The Food and Drug Administration has expanded the indication for Orkambi (lumacaftor/ivacaftor) to include patients who are aged as young as 2 years with cystic fibrosis (CF), according to its manufacturer, Vertex Pharmaceuticals. Specifically, the drug is meant to treat the most common underlying cause of CF – having two copies of the F508del-CFTR mutation – and is the first drug to treat it.

The approval is based on a phase 3, two-part, open-label, multicenter study that assessed various doses in patents aged 2-5 years. The study demonstrated safety and tolerability in that age group equivalent to that seen in older patients. The drug is expected to be available for this age group within 2-4 weeks of this approval.

Available as oral granules in two doses for weight-based dosing (either lumacaftor 100 mg/ivacaftor 125 mg or lumacaftor 150 mg/ivacaftor 188 mg), the compound targets the defective chloride channels responsible for CF; the two halves work together to increase the number of chloride channels on cell surfaces and also improve their function.

Orkambi should be prescribed only for patients with CF who have the dual F508del-CFTR mutation; it is not indicated for other types of CF. Patients should not take this drug if they are taking drugs such as rifampin, phenytoin, triazolam, or cyclosporine because of possible drug interactions. It can also lead to worsening liver function and elevated blood liver enzymes, increased blood pressure, or cataracts. The most common side effects include breathing problems, nausea, fatigue, and rash. Full prescribing information is available on the FDA website.
 

[email protected]

The Food and Drug Administration has expanded the indication for Orkambi (lumacaftor/ivacaftor) to include patients who are aged as young as 2 years with cystic fibrosis (CF), according to its manufacturer, Vertex Pharmaceuticals. Specifically, the drug is meant to treat the most common underlying cause of CF – having two copies of the F508del-CFTR mutation – and is the first drug to treat it.

The approval is based on a phase 3, two-part, open-label, multicenter study that assessed various doses in patents aged 2-5 years. The study demonstrated safety and tolerability in that age group equivalent to that seen in older patients. The drug is expected to be available for this age group within 2-4 weeks of this approval.

Available as oral granules in two doses for weight-based dosing (either lumacaftor 100 mg/ivacaftor 125 mg or lumacaftor 150 mg/ivacaftor 188 mg), the compound targets the defective chloride channels responsible for CF; the two halves work together to increase the number of chloride channels on cell surfaces and also improve their function.

Orkambi should be prescribed only for patients with CF who have the dual F508del-CFTR mutation; it is not indicated for other types of CF. Patients should not take this drug if they are taking drugs such as rifampin, phenytoin, triazolam, or cyclosporine because of possible drug interactions. It can also lead to worsening liver function and elevated blood liver enzymes, increased blood pressure, or cataracts. The most common side effects include breathing problems, nausea, fatigue, and rash. Full prescribing information is available on the FDA website.
 

[email protected]

The Food and Drug Administration has issued a safety alert warning against long-term use of azithromycin to prevent bronchiolitis obliterans syndrome in patients with blood or lymph node cancers who have received donor stem cell transplants.

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The Food and Drug Administration has issued a safety alert warning against long-term use of azithromycin to prevent bronchiolitis obliterans syndrome in patients with blood or lymph node cancers who have received donor stem cell transplants.

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The Food and Drug Administration has issued a safety alert warning against long-term use of azithromycin to prevent bronchiolitis obliterans syndrome in patients with blood or lymph node cancers who have received donor stem cell transplants.

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