User login
Recombinant coagulation factor IX approved for hemophilia B
Nonacog beta pegol (N9-GP, Rebinyn®), a recombinant, GlycoPEGylated coagulation factor IX, has been approved by the US Food and Drug Administration (FDA) for on-demand treatment and control of bleeding episodes in adults and children with hemophilia B.
It is also indicated for the perioperative management of bleeding in these individuals. However, it is not indicated for routine prophylaxis or for immune tolerance induction.
NovoNordisk, the manufacturer of the drug, expects the launch of the coagulation factor in the United States to take place in the first half of 2018.
N9-GP earlier this year had been recommended for marketing authorization by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) as Refixia.® On June 6 it was actually granted marketing authorization, which covers all 28 European Union member states.
The FDA based its approval on the efficacy and safety evaluation of 115 previously treated patients across the four paradigmTM clinical trials. Results from the paradigm 4 trial were published in Thrombosis Research in May 2016.
Of 597 new bleeding episodes in 79 of 105 patients, 551 (93%) were resolved successfully with good or excellent ratings by the patients or study site investigator. Forty (7%) were rated as moderate or poor.
Most (87%) were resolved with 1 injection, 60 (10%) with 2 injections, and 16 (3%) with more than 2 injections.
The median dose to treat a bleeding episode was 42.3 IU/kg.
On-demand treatment had a success rate of 95%, with 120 (84%) of the 143 bleeds treated with one injection.
For perioperative management, N9-GP was rated as excellent or good for 13 surgeries, for a success rate of 100%.
Patients received a preoperative dose of 80 IU/kg. No patient required additional doses on the day of surgery.
During the postoperative period, patients required additional 40 IU/kg doses. The mean total consumption of factor in the pre- and postoperative period was 241 IU/kg (range, 8 – 460 IU/kg.
No unexpected postoperative bleeding occurred.
For full prescribing information, see the package insert. 
Nonacog beta pegol (N9-GP, Rebinyn®), a recombinant, GlycoPEGylated coagulation factor IX, has been approved by the US Food and Drug Administration (FDA) for on-demand treatment and control of bleeding episodes in adults and children with hemophilia B.
It is also indicated for the perioperative management of bleeding in these individuals. However, it is not indicated for routine prophylaxis or for immune tolerance induction.
NovoNordisk, the manufacturer of the drug, expects the launch of the coagulation factor in the United States to take place in the first half of 2018.
N9-GP earlier this year had been recommended for marketing authorization by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) as Refixia.® On June 6 it was actually granted marketing authorization, which covers all 28 European Union member states.
The FDA based its approval on the efficacy and safety evaluation of 115 previously treated patients across the four paradigmTM clinical trials. Results from the paradigm 4 trial were published in Thrombosis Research in May 2016.
Of 597 new bleeding episodes in 79 of 105 patients, 551 (93%) were resolved successfully with good or excellent ratings by the patients or study site investigator. Forty (7%) were rated as moderate or poor.
Most (87%) were resolved with 1 injection, 60 (10%) with 2 injections, and 16 (3%) with more than 2 injections.
The median dose to treat a bleeding episode was 42.3 IU/kg.
On-demand treatment had a success rate of 95%, with 120 (84%) of the 143 bleeds treated with one injection.
For perioperative management, N9-GP was rated as excellent or good for 13 surgeries, for a success rate of 100%.
Patients received a preoperative dose of 80 IU/kg. No patient required additional doses on the day of surgery.
During the postoperative period, patients required additional 40 IU/kg doses. The mean total consumption of factor in the pre- and postoperative period was 241 IU/kg (range, 8 – 460 IU/kg.
No unexpected postoperative bleeding occurred.
For full prescribing information, see the package insert.
Nonacog beta pegol (N9-GP, Rebinyn®), a recombinant, GlycoPEGylated coagulation factor IX, has been approved by the US Food and Drug Administration (FDA) for on-demand treatment and control of bleeding episodes in adults and children with hemophilia B.
It is also indicated for the perioperative management of bleeding in these individuals. However, it is not indicated for routine prophylaxis or for immune tolerance induction.
NovoNordisk, the manufacturer of the drug, expects the launch of the coagulation factor in the United States to take place in the first half of 2018.
N9-GP earlier this year had been recommended for marketing authorization by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) as Refixia.® On June 6 it was actually granted marketing authorization, which covers all 28 European Union member states.
The FDA based its approval on the efficacy and safety evaluation of 115 previously treated patients across the four paradigmTM clinical trials. Results from the paradigm 4 trial were published in Thrombosis Research in May 2016.
Of 597 new bleeding episodes in 79 of 105 patients, 551 (93%) were resolved successfully with good or excellent ratings by the patients or study site investigator. Forty (7%) were rated as moderate or poor.
Most (87%) were resolved with 1 injection, 60 (10%) with 2 injections, and 16 (3%) with more than 2 injections.
The median dose to treat a bleeding episode was 42.3 IU/kg.
On-demand treatment had a success rate of 95%, with 120 (84%) of the 143 bleeds treated with one injection.
For perioperative management, N9-GP was rated as excellent or good for 13 surgeries, for a success rate of 100%.
Patients received a preoperative dose of 80 IU/kg. No patient required additional doses on the day of surgery.
During the postoperative period, patients required additional 40 IU/kg doses. The mean total consumption of factor in the pre- and postoperative period was 241 IU/kg (range, 8 – 460 IU/kg.
No unexpected postoperative bleeding occurred.
For full prescribing information, see the package insert.
EMA recommends orphan designation for hemophilia B product
The European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products has issued a positive opinion recommending orphan designation for CB 2679d/ISU304 for the treatment of hemophilia B.
CB 2679d is a coagulation factor IX variant that has demonstrated, in preclinical studies, the potential to normalize factor IX levels via a daily subcutaneous injection.
The product is being developed by Catalyst Biosciences and ISU Abxis. ISU Abxis plans to initiate a phase 1/2 study of CB 2679d in individuals with severe hemophilia B this month in South Korea.
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
The EMA’s Committee for Orphan Medicinal Products adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the European Commission for a final decision. The commission typically makes a decision within 30 days of the submission.
The European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products has issued a positive opinion recommending orphan designation for CB 2679d/ISU304 for the treatment of hemophilia B.
CB 2679d is a coagulation factor IX variant that has demonstrated, in preclinical studies, the potential to normalize factor IX levels via a daily subcutaneous injection.
The product is being developed by Catalyst Biosciences and ISU Abxis. ISU Abxis plans to initiate a phase 1/2 study of CB 2679d in individuals with severe hemophilia B this month in South Korea.
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
The EMA’s Committee for Orphan Medicinal Products adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the European Commission for a final decision. The commission typically makes a decision within 30 days of the submission.
The European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products has issued a positive opinion recommending orphan designation for CB 2679d/ISU304 for the treatment of hemophilia B.
CB 2679d is a coagulation factor IX variant that has demonstrated, in preclinical studies, the potential to normalize factor IX levels via a daily subcutaneous injection.
The product is being developed by Catalyst Biosciences and ISU Abxis. ISU Abxis plans to initiate a phase 1/2 study of CB 2679d in individuals with severe hemophilia B this month in South Korea.
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
The EMA’s Committee for Orphan Medicinal Products adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the European Commission for a final decision. The commission typically makes a decision within 30 days of the submission.
BLA for CAR T-cell therapy granted priority review
The US Food and Drug Administration (FDA) has accepted for priority review the biologics license application (BLA) for axicabtagene ciloleucel (formerly KTE-C19), a chimeric antigen receptor (CAR) T-cell therapy.
Kite Pharma, Inc. is seeking approval for axicabtagene ciloleucel as a treatment for patients with relapsed or refractory, aggressive non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant.
The FDA grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The agency’s goal is to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The FDA has set a review deadline of November 29, 2017, for the axicabtagene ciloleucel BLA.
Axicabtagene ciloleucel also has breakthrough therapy designation from the FDA as a treatment for diffuse large B-cell lymphoma, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma.
ZUMA-1 trial
The BLA for axicabtagene ciloleucel is supported by data from the phase 2 ZUMA-1 trial, which enrolled 111 patients with relapsed/refractory B-cell NHL.
After a single infusion of axicabtagene ciloleucel, the objective response rate was 82%. At a median follow-up of 8.7 months, 44% of patients were still in response, which included 39% of patients in complete response.
The most common grade 3 or higher adverse events were anemia (43%), neutropenia (39%), decreased neutrophil count (32%), febrile neutropenia (31%), decreased white blood cell count (29%), thrombocytopenia (24%), encephalopathy (21%), and decreased lymphocyte count (20%).
There were 3 deaths during the trial that were not due to disease progression. Two of these deaths were deemed related to axicabtagene ciloleucel.
The US Food and Drug Administration (FDA) has accepted for priority review the biologics license application (BLA) for axicabtagene ciloleucel (formerly KTE-C19), a chimeric antigen receptor (CAR) T-cell therapy.
Kite Pharma, Inc. is seeking approval for axicabtagene ciloleucel as a treatment for patients with relapsed or refractory, aggressive non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant.
The FDA grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The agency’s goal is to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The FDA has set a review deadline of November 29, 2017, for the axicabtagene ciloleucel BLA.
Axicabtagene ciloleucel also has breakthrough therapy designation from the FDA as a treatment for diffuse large B-cell lymphoma, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma.
ZUMA-1 trial
The BLA for axicabtagene ciloleucel is supported by data from the phase 2 ZUMA-1 trial, which enrolled 111 patients with relapsed/refractory B-cell NHL.
After a single infusion of axicabtagene ciloleucel, the objective response rate was 82%. At a median follow-up of 8.7 months, 44% of patients were still in response, which included 39% of patients in complete response.
The most common grade 3 or higher adverse events were anemia (43%), neutropenia (39%), decreased neutrophil count (32%), febrile neutropenia (31%), decreased white blood cell count (29%), thrombocytopenia (24%), encephalopathy (21%), and decreased lymphocyte count (20%).
There were 3 deaths during the trial that were not due to disease progression. Two of these deaths were deemed related to axicabtagene ciloleucel.
The US Food and Drug Administration (FDA) has accepted for priority review the biologics license application (BLA) for axicabtagene ciloleucel (formerly KTE-C19), a chimeric antigen receptor (CAR) T-cell therapy.
Kite Pharma, Inc. is seeking approval for axicabtagene ciloleucel as a treatment for patients with relapsed or refractory, aggressive non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant.
The FDA grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The agency’s goal is to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The FDA has set a review deadline of November 29, 2017, for the axicabtagene ciloleucel BLA.
Axicabtagene ciloleucel also has breakthrough therapy designation from the FDA as a treatment for diffuse large B-cell lymphoma, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma.
ZUMA-1 trial
The BLA for axicabtagene ciloleucel is supported by data from the phase 2 ZUMA-1 trial, which enrolled 111 patients with relapsed/refractory B-cell NHL.
After a single infusion of axicabtagene ciloleucel, the objective response rate was 82%. At a median follow-up of 8.7 months, 44% of patients were still in response, which included 39% of patients in complete response.
The most common grade 3 or higher adverse events were anemia (43%), neutropenia (39%), decreased neutrophil count (32%), febrile neutropenia (31%), decreased white blood cell count (29%), thrombocytopenia (24%), encephalopathy (21%), and decreased lymphocyte count (20%).
There were 3 deaths during the trial that were not due to disease progression. Two of these deaths were deemed related to axicabtagene ciloleucel.
AstraZeneca recalls lot of Brilinta in US
AstraZeneca has announced a voluntary recall of 1 lot of professional (physician) sample bottles containing 8 tablets of Brilinta® (ticagrelor).
This recall follows a report that a professional sample bottle containing 8 tablets of Brilinta 90 mg also contained Zurampic® (lesinurad) 200 mg tablets, which is also manufactured by AstraZeneca.
The company said this precautionary measure is limited to 1 lot of Brilinta (Brilinta lot #JB5047) distributed to physicians in the US between March and April of 2017.
Other forms and dosage strengths of Brilinta, including medicine obtained via US retail or mail order pharmacies, are not affected by this recall. And this recall does not affect Zurampic.
Potential risks
Unintentional dosing with Zurampic has the potential to lead to adverse renal effects, including acute renal failure, which is more common when Zurampic is given alone, as it should be used in combination with a xanthine oxidase inhibitor.
Missed doses of Brilinta increase the risk of heart attack and stroke. People with a stent who miss doses of Brilinta have a higher risk of stent thrombosis, heart attack, and death. Patients should not stop taking Brilinta without talking to their prescribing doctor.
To date, AstraZeneca has not received any reports of adverse events related to this recall.
Next steps
AstraZeneca is notifying physicians by recall letter and is arranging for the return of all recalled products. Consumers who have medicine that is being recalled should contact their physician.
Consumers with questions regarding this recall can contact the AstraZeneca Information Center at 1-800-236-9933 between the hours of 8 am and 6 pm (Eastern time) Monday to Friday, excluding holidays.
Consumers should contact their physician or healthcare provider if they have experienced any problems that may be related to taking or using Brilinta.
Adverse reactions or quality problems related to Brilinta may also be reported to the FDA’s MedWatch Adverse Event Reporting Program.
About Brilinta and Zurampic
Brilinta is intended for use to reduce the rate of cardiovascular death, heart attack, and stroke in patients with acute coronary syndrome or a history of heart attack.
Brilinta is also intended to reduce the rate of stent thrombosis in patients who have been stented for treatment of acute coronary syndrome.
Zurampic is used together with a xanthine oxidase inhibitor, such as allopurinol or Uloric, in adults with gout who still have a high uric acid level.
Brilinta 90 mg tablets are supplied as a round, biconvex, yellow, film-coated tablet, and imprinted with a “90” above a “T” on one side of the pill.
Zurampic tablets 200 mg are blue in color and elliptical/oval in shape. They are imprinted with “LES200” on one side of the pill.
AstraZeneca has announced a voluntary recall of 1 lot of professional (physician) sample bottles containing 8 tablets of Brilinta® (ticagrelor).
This recall follows a report that a professional sample bottle containing 8 tablets of Brilinta 90 mg also contained Zurampic® (lesinurad) 200 mg tablets, which is also manufactured by AstraZeneca.
The company said this precautionary measure is limited to 1 lot of Brilinta (Brilinta lot #JB5047) distributed to physicians in the US between March and April of 2017.
Other forms and dosage strengths of Brilinta, including medicine obtained via US retail or mail order pharmacies, are not affected by this recall. And this recall does not affect Zurampic.
Potential risks
Unintentional dosing with Zurampic has the potential to lead to adverse renal effects, including acute renal failure, which is more common when Zurampic is given alone, as it should be used in combination with a xanthine oxidase inhibitor.
Missed doses of Brilinta increase the risk of heart attack and stroke. People with a stent who miss doses of Brilinta have a higher risk of stent thrombosis, heart attack, and death. Patients should not stop taking Brilinta without talking to their prescribing doctor.
To date, AstraZeneca has not received any reports of adverse events related to this recall.
Next steps
AstraZeneca is notifying physicians by recall letter and is arranging for the return of all recalled products. Consumers who have medicine that is being recalled should contact their physician.
Consumers with questions regarding this recall can contact the AstraZeneca Information Center at 1-800-236-9933 between the hours of 8 am and 6 pm (Eastern time) Monday to Friday, excluding holidays.
Consumers should contact their physician or healthcare provider if they have experienced any problems that may be related to taking or using Brilinta.
Adverse reactions or quality problems related to Brilinta may also be reported to the FDA’s MedWatch Adverse Event Reporting Program.
About Brilinta and Zurampic
Brilinta is intended for use to reduce the rate of cardiovascular death, heart attack, and stroke in patients with acute coronary syndrome or a history of heart attack.
Brilinta is also intended to reduce the rate of stent thrombosis in patients who have been stented for treatment of acute coronary syndrome.
Zurampic is used together with a xanthine oxidase inhibitor, such as allopurinol or Uloric, in adults with gout who still have a high uric acid level.
Brilinta 90 mg tablets are supplied as a round, biconvex, yellow, film-coated tablet, and imprinted with a “90” above a “T” on one side of the pill.
Zurampic tablets 200 mg are blue in color and elliptical/oval in shape. They are imprinted with “LES200” on one side of the pill.
AstraZeneca has announced a voluntary recall of 1 lot of professional (physician) sample bottles containing 8 tablets of Brilinta® (ticagrelor).
This recall follows a report that a professional sample bottle containing 8 tablets of Brilinta 90 mg also contained Zurampic® (lesinurad) 200 mg tablets, which is also manufactured by AstraZeneca.
The company said this precautionary measure is limited to 1 lot of Brilinta (Brilinta lot #JB5047) distributed to physicians in the US between March and April of 2017.
Other forms and dosage strengths of Brilinta, including medicine obtained via US retail or mail order pharmacies, are not affected by this recall. And this recall does not affect Zurampic.
Potential risks
Unintentional dosing with Zurampic has the potential to lead to adverse renal effects, including acute renal failure, which is more common when Zurampic is given alone, as it should be used in combination with a xanthine oxidase inhibitor.
Missed doses of Brilinta increase the risk of heart attack and stroke. People with a stent who miss doses of Brilinta have a higher risk of stent thrombosis, heart attack, and death. Patients should not stop taking Brilinta without talking to their prescribing doctor.
To date, AstraZeneca has not received any reports of adverse events related to this recall.
Next steps
AstraZeneca is notifying physicians by recall letter and is arranging for the return of all recalled products. Consumers who have medicine that is being recalled should contact their physician.
Consumers with questions regarding this recall can contact the AstraZeneca Information Center at 1-800-236-9933 between the hours of 8 am and 6 pm (Eastern time) Monday to Friday, excluding holidays.
Consumers should contact their physician or healthcare provider if they have experienced any problems that may be related to taking or using Brilinta.
Adverse reactions or quality problems related to Brilinta may also be reported to the FDA’s MedWatch Adverse Event Reporting Program.
About Brilinta and Zurampic
Brilinta is intended for use to reduce the rate of cardiovascular death, heart attack, and stroke in patients with acute coronary syndrome or a history of heart attack.
Brilinta is also intended to reduce the rate of stent thrombosis in patients who have been stented for treatment of acute coronary syndrome.
Zurampic is used together with a xanthine oxidase inhibitor, such as allopurinol or Uloric, in adults with gout who still have a high uric acid level.
Brilinta 90 mg tablets are supplied as a round, biconvex, yellow, film-coated tablet, and imprinted with a “90” above a “T” on one side of the pill.
Zurampic tablets 200 mg are blue in color and elliptical/oval in shape. They are imprinted with “LES200” on one side of the pill.
EC grants drug orphan designation for AML
The European Commission (EC) has granted orphan designation to GMI-1271 for the treatment of acute myeloid leukemia (AML).
GMI-1271 is an E-selectin antagonist being developed by GlycoMimetics, Inc.
The product also has orphan designation, fast track designation, and breakthrough therapy designation in the US.
GMI-1271 is currently being evaluated in a phase 1/2 trial of patients with relapsed or refractory AML and patients age 60 and older with newly diagnosed AML.
The patients are receiving GM-1271 in combination with chemotherapy. The relapsed/refractory group is receiving mitoxantrone, etoposide, and cytarabine. The newly diagnosed patients are receiving cytarabine and idarubicin (7+3).
GlycoMimetics plans to present data from this trial at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting as abstracts 2520 and 2560.
The company also plans to present the research at the 22nd Congress of the European Hematology Association (EHA) as abstracts P547 and P203.
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.
The European Medicines Agency adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the EC for a final decision. The EC typically makes a decision within 30 days of that submission.
The European Commission (EC) has granted orphan designation to GMI-1271 for the treatment of acute myeloid leukemia (AML).
GMI-1271 is an E-selectin antagonist being developed by GlycoMimetics, Inc.
The product also has orphan designation, fast track designation, and breakthrough therapy designation in the US.
GMI-1271 is currently being evaluated in a phase 1/2 trial of patients with relapsed or refractory AML and patients age 60 and older with newly diagnosed AML.
The patients are receiving GM-1271 in combination with chemotherapy. The relapsed/refractory group is receiving mitoxantrone, etoposide, and cytarabine. The newly diagnosed patients are receiving cytarabine and idarubicin (7+3).
GlycoMimetics plans to present data from this trial at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting as abstracts 2520 and 2560.
The company also plans to present the research at the 22nd Congress of the European Hematology Association (EHA) as abstracts P547 and P203.
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.
The European Medicines Agency adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the EC for a final decision. The EC typically makes a decision within 30 days of that submission.
The European Commission (EC) has granted orphan designation to GMI-1271 for the treatment of acute myeloid leukemia (AML).
GMI-1271 is an E-selectin antagonist being developed by GlycoMimetics, Inc.
The product also has orphan designation, fast track designation, and breakthrough therapy designation in the US.
GMI-1271 is currently being evaluated in a phase 1/2 trial of patients with relapsed or refractory AML and patients age 60 and older with newly diagnosed AML.
The patients are receiving GM-1271 in combination with chemotherapy. The relapsed/refractory group is receiving mitoxantrone, etoposide, and cytarabine. The newly diagnosed patients are receiving cytarabine and idarubicin (7+3).
GlycoMimetics plans to present data from this trial at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting as abstracts 2520 and 2560.
The company also plans to present the research at the 22nd Congress of the European Hematology Association (EHA) as abstracts P547 and P203.
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.
The European Medicines Agency adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the EC for a final decision. The EC typically makes a decision within 30 days of that submission.
EC grants drug orphan designation for CTCL
The European Commission (EC) has granted orphan designation to MRG-106 for the treatment of cutaneous T-cell lymphoma (CTCL).
MRG-106 is a locked nucleic acid-modified oligonucleotide inhibitor of miR-155-5p.
miRagen Therapeutics, Inc., the company developing MRG-106, is currently testing the drug in a phase 1 trial of CTCL patients.
Early results from this trial were presented at the 2016 ASH Annual Meeting.
Researchers presented results in 6 patients with stage I-III mycosis fungoides.
The patients received 4 or 5 intratumoral injections of MRG-106 (at 75 mg) over 2 weeks. Four patients received saline injections in a second lesion on the same schedule.
There were 3 adverse events related to MRG-106—pain during injection, burning sensation during injection, and tingling at the injection site.
Adverse events considered possibly related to MRG-106 were pruritus, erythema, skin inflammation, sore on hand, nausea, decrease in white blood cells, neutropenia, and prolonged partial thromboplastin time.
One patient was taken off the trial due to rapid disease progression. The other 5 patients completed the dosing period.
All 5 patients had a reduction in the baseline Composite Assessment of Index Lesion Severity score in MRG-106-treated and saline-treated lesions.
The average maximal reduction was 55% (range, 33% to 77%) in MRG-106-treated lesions and 39% (range, 13% to 75%) in saline-treated lesions.
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.
The European Medicines Agency adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the EC for a final decision. The EC typically makes a decision within 30 days of that submission.
The European Commission (EC) has granted orphan designation to MRG-106 for the treatment of cutaneous T-cell lymphoma (CTCL).
MRG-106 is a locked nucleic acid-modified oligonucleotide inhibitor of miR-155-5p.
miRagen Therapeutics, Inc., the company developing MRG-106, is currently testing the drug in a phase 1 trial of CTCL patients.
Early results from this trial were presented at the 2016 ASH Annual Meeting.
Researchers presented results in 6 patients with stage I-III mycosis fungoides.
The patients received 4 or 5 intratumoral injections of MRG-106 (at 75 mg) over 2 weeks. Four patients received saline injections in a second lesion on the same schedule.
There were 3 adverse events related to MRG-106—pain during injection, burning sensation during injection, and tingling at the injection site.
Adverse events considered possibly related to MRG-106 were pruritus, erythema, skin inflammation, sore on hand, nausea, decrease in white blood cells, neutropenia, and prolonged partial thromboplastin time.
One patient was taken off the trial due to rapid disease progression. The other 5 patients completed the dosing period.
All 5 patients had a reduction in the baseline Composite Assessment of Index Lesion Severity score in MRG-106-treated and saline-treated lesions.
The average maximal reduction was 55% (range, 33% to 77%) in MRG-106-treated lesions and 39% (range, 13% to 75%) in saline-treated lesions.
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.
The European Medicines Agency adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the EC for a final decision. The EC typically makes a decision within 30 days of that submission.
The European Commission (EC) has granted orphan designation to MRG-106 for the treatment of cutaneous T-cell lymphoma (CTCL).
MRG-106 is a locked nucleic acid-modified oligonucleotide inhibitor of miR-155-5p.
miRagen Therapeutics, Inc., the company developing MRG-106, is currently testing the drug in a phase 1 trial of CTCL patients.
Early results from this trial were presented at the 2016 ASH Annual Meeting.
Researchers presented results in 6 patients with stage I-III mycosis fungoides.
The patients received 4 or 5 intratumoral injections of MRG-106 (at 75 mg) over 2 weeks. Four patients received saline injections in a second lesion on the same schedule.
There were 3 adverse events related to MRG-106—pain during injection, burning sensation during injection, and tingling at the injection site.
Adverse events considered possibly related to MRG-106 were pruritus, erythema, skin inflammation, sore on hand, nausea, decrease in white blood cells, neutropenia, and prolonged partial thromboplastin time.
One patient was taken off the trial due to rapid disease progression. The other 5 patients completed the dosing period.
All 5 patients had a reduction in the baseline Composite Assessment of Index Lesion Severity score in MRG-106-treated and saline-treated lesions.
The average maximal reduction was 55% (range, 33% to 77%) in MRG-106-treated lesions and 39% (range, 13% to 75%) in saline-treated lesions.
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval.
The designation also provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase and direct access to the centralized authorization procedure.
The European Medicines Agency adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the EC for a final decision. The EC typically makes a decision within 30 days of that submission.
EMA recommends orphan designation for AML drug
The European Medicines Agency (EMA) has recommended orphan designation for Actimab-A, a product intended to treat patients with newly diagnosed acute myeloid leukemia (AML) who are over the age of 60 and are ineligible for standard induction therapy.
Actimab-A targets CD33, a protein expressed on the surface of AML cells, via the monoclonal antibody, HuM195, which carries the cytotoxic radioisotope actinium-225 to the AML cells.
Actinium Pharmaceuticals, Inc., the company developing Actimab-A, is testing the drug in a phase 2 trial.
Results from a phase 1 trial of the drug were presented at the 2016 ASH Annual Meeting.
At that time, researchers reported results in 18 patients who had been newly diagnosed with AML and were age 60 and older. Their median age was 77 (range, 68-87).
The patients received Actimab-A in combination with low-dose cytarabine. Actimab-A was given at 0.5 μCi/kg/fraction (n=3), 1 μCi/kg/fraction (n=6), 1.5 μCi/kg/fraction (n=3), or 2 μCi/kg/fraction (n=6).
Two patients experienced dose-limiting toxicities. Both had grade 4 thrombocytopenia with marrow aplasia for more than 6 weeks after therapy. One patient was in the 1 µCi/kg/fraction cohort, and the other was in the 2 µCi/kg/fraction cohort.
The maximum-tolerated dose was not reached, but 2 µCi/kg/fraction was chosen as the phase 2 dose.
Grade 3/4 toxicities included neutropenia (n=5), thrombocytopenia (n=9), febrile neutropenia (n=6), pneumonia (n=5), other infections (n=3), atrial fibrillation/syncope (n=1), transient creatinine increase (n=1), generalized fatigue (n=1), hypokalemia (n=1), mucositis (n=1), and rectal hemorrhage (n=1).
Twenty-eight percent of patients (5/18) had objective responses to treatment. Two patients achieved a complete response (CR), 1 had a CR with incomplete platelet recovery, and 2 had a CR with incomplete marrow recovery.
The median duration of response was 9.1 months (range, 4.1-16.9).
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval. The designation also provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
The EMA adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the European Commission for a final decision. The European Commission typically makes a decision within 30 days.
The European Medicines Agency (EMA) has recommended orphan designation for Actimab-A, a product intended to treat patients with newly diagnosed acute myeloid leukemia (AML) who are over the age of 60 and are ineligible for standard induction therapy.
Actimab-A targets CD33, a protein expressed on the surface of AML cells, via the monoclonal antibody, HuM195, which carries the cytotoxic radioisotope actinium-225 to the AML cells.
Actinium Pharmaceuticals, Inc., the company developing Actimab-A, is testing the drug in a phase 2 trial.
Results from a phase 1 trial of the drug were presented at the 2016 ASH Annual Meeting.
At that time, researchers reported results in 18 patients who had been newly diagnosed with AML and were age 60 and older. Their median age was 77 (range, 68-87).
The patients received Actimab-A in combination with low-dose cytarabine. Actimab-A was given at 0.5 μCi/kg/fraction (n=3), 1 μCi/kg/fraction (n=6), 1.5 μCi/kg/fraction (n=3), or 2 μCi/kg/fraction (n=6).
Two patients experienced dose-limiting toxicities. Both had grade 4 thrombocytopenia with marrow aplasia for more than 6 weeks after therapy. One patient was in the 1 µCi/kg/fraction cohort, and the other was in the 2 µCi/kg/fraction cohort.
The maximum-tolerated dose was not reached, but 2 µCi/kg/fraction was chosen as the phase 2 dose.
Grade 3/4 toxicities included neutropenia (n=5), thrombocytopenia (n=9), febrile neutropenia (n=6), pneumonia (n=5), other infections (n=3), atrial fibrillation/syncope (n=1), transient creatinine increase (n=1), generalized fatigue (n=1), hypokalemia (n=1), mucositis (n=1), and rectal hemorrhage (n=1).
Twenty-eight percent of patients (5/18) had objective responses to treatment. Two patients achieved a complete response (CR), 1 had a CR with incomplete platelet recovery, and 2 had a CR with incomplete marrow recovery.
The median duration of response was 9.1 months (range, 4.1-16.9).
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval. The designation also provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
The EMA adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the European Commission for a final decision. The European Commission typically makes a decision within 30 days.
The European Medicines Agency (EMA) has recommended orphan designation for Actimab-A, a product intended to treat patients with newly diagnosed acute myeloid leukemia (AML) who are over the age of 60 and are ineligible for standard induction therapy.
Actimab-A targets CD33, a protein expressed on the surface of AML cells, via the monoclonal antibody, HuM195, which carries the cytotoxic radioisotope actinium-225 to the AML cells.
Actinium Pharmaceuticals, Inc., the company developing Actimab-A, is testing the drug in a phase 2 trial.
Results from a phase 1 trial of the drug were presented at the 2016 ASH Annual Meeting.
At that time, researchers reported results in 18 patients who had been newly diagnosed with AML and were age 60 and older. Their median age was 77 (range, 68-87).
The patients received Actimab-A in combination with low-dose cytarabine. Actimab-A was given at 0.5 μCi/kg/fraction (n=3), 1 μCi/kg/fraction (n=6), 1.5 μCi/kg/fraction (n=3), or 2 μCi/kg/fraction (n=6).
Two patients experienced dose-limiting toxicities. Both had grade 4 thrombocytopenia with marrow aplasia for more than 6 weeks after therapy. One patient was in the 1 µCi/kg/fraction cohort, and the other was in the 2 µCi/kg/fraction cohort.
The maximum-tolerated dose was not reached, but 2 µCi/kg/fraction was chosen as the phase 2 dose.
Grade 3/4 toxicities included neutropenia (n=5), thrombocytopenia (n=9), febrile neutropenia (n=6), pneumonia (n=5), other infections (n=3), atrial fibrillation/syncope (n=1), transient creatinine increase (n=1), generalized fatigue (n=1), hypokalemia (n=1), mucositis (n=1), and rectal hemorrhage (n=1).
Twenty-eight percent of patients (5/18) had objective responses to treatment. Two patients achieved a complete response (CR), 1 had a CR with incomplete platelet recovery, and 2 had a CR with incomplete marrow recovery.
The median duration of response was 9.1 months (range, 4.1-16.9).
About orphan designation
Orphan designation provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.
Orphan designation provides a 10-year period of marketing exclusivity if the drug receives regulatory approval. The designation also provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized authorization procedure.
The EMA adopts an opinion on the granting of orphan drug designation, and that opinion is submitted to the European Commission for a final decision. The European Commission typically makes a decision within 30 days.
FDA approves new formulation of deferasirox
The US Food and Drug Administration (FDA) has approved a new formulation of deferasirox known as Jadenu Sprinkle granules.
The granules are approved for use in the same population as Jadenu film-coated tablets.
Both formulations of Jadenu have accelerated approval from the FDA for the treatment of chronic iron overload due to blood transfusions in patients age 2 and older.
The formulations also have accelerated FDA approval for the treatment of chronic iron overload in patients age 10 and older with non-transfusion-dependent-thalassemia and a liver iron concentration of at least 5 mg Fe per gram of dry weight and a serum ferritin greater than 300 mcg/L.
Continued FDA approval for Jadenu in these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
Jadenu Sprinkle granules are intended for patients who have difficulty swallowing whole tablets. The granules can be sprinkled over soft foods (eg, yogurt or applesauce) prior to consumption.
Like Jadenu film-coated tablets, Jadenu Sprinkle granules are available in 3 strengths—90 mg, 180 mg, and 360 mg.
Both formulations of Jadenu are products of Novartis. For more details on Jadenu, see the prescribing information.
The US Food and Drug Administration (FDA) has approved a new formulation of deferasirox known as Jadenu Sprinkle granules.
The granules are approved for use in the same population as Jadenu film-coated tablets.
Both formulations of Jadenu have accelerated approval from the FDA for the treatment of chronic iron overload due to blood transfusions in patients age 2 and older.
The formulations also have accelerated FDA approval for the treatment of chronic iron overload in patients age 10 and older with non-transfusion-dependent-thalassemia and a liver iron concentration of at least 5 mg Fe per gram of dry weight and a serum ferritin greater than 300 mcg/L.
Continued FDA approval for Jadenu in these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
Jadenu Sprinkle granules are intended for patients who have difficulty swallowing whole tablets. The granules can be sprinkled over soft foods (eg, yogurt or applesauce) prior to consumption.
Like Jadenu film-coated tablets, Jadenu Sprinkle granules are available in 3 strengths—90 mg, 180 mg, and 360 mg.
Both formulations of Jadenu are products of Novartis. For more details on Jadenu, see the prescribing information.
The US Food and Drug Administration (FDA) has approved a new formulation of deferasirox known as Jadenu Sprinkle granules.
The granules are approved for use in the same population as Jadenu film-coated tablets.
Both formulations of Jadenu have accelerated approval from the FDA for the treatment of chronic iron overload due to blood transfusions in patients age 2 and older.
The formulations also have accelerated FDA approval for the treatment of chronic iron overload in patients age 10 and older with non-transfusion-dependent-thalassemia and a liver iron concentration of at least 5 mg Fe per gram of dry weight and a serum ferritin greater than 300 mcg/L.
Continued FDA approval for Jadenu in these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
Jadenu Sprinkle granules are intended for patients who have difficulty swallowing whole tablets. The granules can be sprinkled over soft foods (eg, yogurt or applesauce) prior to consumption.
Like Jadenu film-coated tablets, Jadenu Sprinkle granules are available in 3 strengths—90 mg, 180 mg, and 360 mg.
Both formulations of Jadenu are products of Novartis. For more details on Jadenu, see the prescribing information.
First generic version of clofarabine available in US
Clofarabine Injection, the first-to-market generic version of Sanofi Genzyme’s Clolar, is now available in the US.
The generic, a product of Fresenius Kabi, is available as a single dose vial containing 20 mg per 20 mL clofarabine.
Clofarabine is a purine nucleoside metabolic inhibitor indicated for the treatment of patients ages 1 to 21 with relapsed or refractory acute lymphoblastic leukemia (ALL) who received at least 2 prior treatment regimens.
Clolar was granted accelerated approval for this indication in the US in 2004.
The approval was based on response rates observed in ALL patients. There are no trials verifying that clofarabine confers improvement in survival or disease-related symptoms in ALL patients.
Clofarabine was assessed in a single-arm, phase 2 trial of 61 pediatric patients with relapsed/refractory ALL.
The patients’ median age was 12 (range, 1 to 20 years), and their median number of prior treatment regimens was 3 (range, 2 to 6).
The patients received clofarabine at 52 mg/m2 intravenously over 2 hours daily for 5 days, every 2 to 6 weeks.
The overall response rate was 30%. Seven patient achieved a complete response (CR), 5 had a CR without platelet recovery, and 6 patients had a partial response.
The median duration of CR in patients who did not go on to hematopoietic stem cell transplant was 6 weeks.
The most common grade 3 or higher adverse events were febrile neutropenia, anorexia, hypotension, and nausea.
These results were published in the Journal of Clinical Oncology in 2006.
Clofarabine Injection, the first-to-market generic version of Sanofi Genzyme’s Clolar, is now available in the US.
The generic, a product of Fresenius Kabi, is available as a single dose vial containing 20 mg per 20 mL clofarabine.
Clofarabine is a purine nucleoside metabolic inhibitor indicated for the treatment of patients ages 1 to 21 with relapsed or refractory acute lymphoblastic leukemia (ALL) who received at least 2 prior treatment regimens.
Clolar was granted accelerated approval for this indication in the US in 2004.
The approval was based on response rates observed in ALL patients. There are no trials verifying that clofarabine confers improvement in survival or disease-related symptoms in ALL patients.
Clofarabine was assessed in a single-arm, phase 2 trial of 61 pediatric patients with relapsed/refractory ALL.
The patients’ median age was 12 (range, 1 to 20 years), and their median number of prior treatment regimens was 3 (range, 2 to 6).
The patients received clofarabine at 52 mg/m2 intravenously over 2 hours daily for 5 days, every 2 to 6 weeks.
The overall response rate was 30%. Seven patient achieved a complete response (CR), 5 had a CR without platelet recovery, and 6 patients had a partial response.
The median duration of CR in patients who did not go on to hematopoietic stem cell transplant was 6 weeks.
The most common grade 3 or higher adverse events were febrile neutropenia, anorexia, hypotension, and nausea.
These results were published in the Journal of Clinical Oncology in 2006.
Clofarabine Injection, the first-to-market generic version of Sanofi Genzyme’s Clolar, is now available in the US.
The generic, a product of Fresenius Kabi, is available as a single dose vial containing 20 mg per 20 mL clofarabine.
Clofarabine is a purine nucleoside metabolic inhibitor indicated for the treatment of patients ages 1 to 21 with relapsed or refractory acute lymphoblastic leukemia (ALL) who received at least 2 prior treatment regimens.
Clolar was granted accelerated approval for this indication in the US in 2004.
The approval was based on response rates observed in ALL patients. There are no trials verifying that clofarabine confers improvement in survival or disease-related symptoms in ALL patients.
Clofarabine was assessed in a single-arm, phase 2 trial of 61 pediatric patients with relapsed/refractory ALL.
The patients’ median age was 12 (range, 1 to 20 years), and their median number of prior treatment regimens was 3 (range, 2 to 6).
The patients received clofarabine at 52 mg/m2 intravenously over 2 hours daily for 5 days, every 2 to 6 weeks.
The overall response rate was 30%. Seven patient achieved a complete response (CR), 5 had a CR without platelet recovery, and 6 patients had a partial response.
The median duration of CR in patients who did not go on to hematopoietic stem cell transplant was 6 weeks.
The most common grade 3 or higher adverse events were febrile neutropenia, anorexia, hypotension, and nausea.
These results were published in the Journal of Clinical Oncology in 2006.
FDA grants priority review to NDA for copanlisib
The US Food and Drug Administration (FDA) has granted priority review to the new drug application (NDA) for copanlisib, an intravenous PI3K inhibitor.
The NDA is for copanlisib as a treatment for patients with relapsed or refractory follicular lymphoma (FL) who have received at least 2 prior therapies.
The FDA grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The agency’s goal is to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The application for copanlisib is supported by data from the CHRONOS-1 trial. This phase 2 trial enrolled 141 patients with relapsed/refractory, indolent non-Hodgkin lymphoma. Most of these patients had FL (n=104).
In all patients, copanlisib produced an objective response rate of 59.2%, with a complete response rate of 12%. The median duration of response exceeded 98 weeks.
In the FL subset, copanlisib produced an overall response rate of 58.7%, with a complete response rate of 14.4%. The median duration of response exceeded 52 weeks.
In the entire cohort, there were 3 deaths considered related to copanlisib.
The most common treatment-related adverse events were transient hyperglycemia (all grades: 49%/grade 3-4: 40%) and hypertension (all grades: 29%/grade 3: 23%).
“Patients with relapsed or refractory follicular lymphoma have a poor prognosis, and new treatment options which are well tolerated and effective are needed to prolong progression-free survival and improve quality of life for these patients,” said Martin Dreyling, MD, a professor at the University of Munich Hospital (Grosshadern) in Germany and lead investigator of the CHRONOS-1 study.
“Based on the CHRONOS-1 results, where copanlisib showed durable efficacy with a manageable and distinct safety profile, the compound may have the potential to address this unmet medical need.”
Data from CHRONOS-1 were presented at the AACR Annual Meeting 2017.
Data from the FL subset of the trial are scheduled to be presented at the 2017 ASCO Annual Meeting in June.
Copanlisib is being developed by Bayer. The compound has fast track and orphan drug designations from the FDA.
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
The FDA’s fast track program is designed to facilitate the development and expedite the review of products intended to treat or prevent serious or life-threatening conditions and address unmet medical need.
Through the fast track program, a product may be eligible for priority review. In addition, the company developing the product may be allowed to submit sections of the NDA or biologic license application on a rolling basis as data become available.
Fast track designation also provides the company with opportunities for more frequent meetings and written communications with the FDA.
The US Food and Drug Administration (FDA) has granted priority review to the new drug application (NDA) for copanlisib, an intravenous PI3K inhibitor.
The NDA is for copanlisib as a treatment for patients with relapsed or refractory follicular lymphoma (FL) who have received at least 2 prior therapies.
The FDA grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The agency’s goal is to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The application for copanlisib is supported by data from the CHRONOS-1 trial. This phase 2 trial enrolled 141 patients with relapsed/refractory, indolent non-Hodgkin lymphoma. Most of these patients had FL (n=104).
In all patients, copanlisib produced an objective response rate of 59.2%, with a complete response rate of 12%. The median duration of response exceeded 98 weeks.
In the FL subset, copanlisib produced an overall response rate of 58.7%, with a complete response rate of 14.4%. The median duration of response exceeded 52 weeks.
In the entire cohort, there were 3 deaths considered related to copanlisib.
The most common treatment-related adverse events were transient hyperglycemia (all grades: 49%/grade 3-4: 40%) and hypertension (all grades: 29%/grade 3: 23%).
“Patients with relapsed or refractory follicular lymphoma have a poor prognosis, and new treatment options which are well tolerated and effective are needed to prolong progression-free survival and improve quality of life for these patients,” said Martin Dreyling, MD, a professor at the University of Munich Hospital (Grosshadern) in Germany and lead investigator of the CHRONOS-1 study.
“Based on the CHRONOS-1 results, where copanlisib showed durable efficacy with a manageable and distinct safety profile, the compound may have the potential to address this unmet medical need.”
Data from CHRONOS-1 were presented at the AACR Annual Meeting 2017.
Data from the FL subset of the trial are scheduled to be presented at the 2017 ASCO Annual Meeting in June.
Copanlisib is being developed by Bayer. The compound has fast track and orphan drug designations from the FDA.
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
The FDA’s fast track program is designed to facilitate the development and expedite the review of products intended to treat or prevent serious or life-threatening conditions and address unmet medical need.
Through the fast track program, a product may be eligible for priority review. In addition, the company developing the product may be allowed to submit sections of the NDA or biologic license application on a rolling basis as data become available.
Fast track designation also provides the company with opportunities for more frequent meetings and written communications with the FDA.
The US Food and Drug Administration (FDA) has granted priority review to the new drug application (NDA) for copanlisib, an intravenous PI3K inhibitor.
The NDA is for copanlisib as a treatment for patients with relapsed or refractory follicular lymphoma (FL) who have received at least 2 prior therapies.
The FDA grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The agency’s goal is to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The application for copanlisib is supported by data from the CHRONOS-1 trial. This phase 2 trial enrolled 141 patients with relapsed/refractory, indolent non-Hodgkin lymphoma. Most of these patients had FL (n=104).
In all patients, copanlisib produced an objective response rate of 59.2%, with a complete response rate of 12%. The median duration of response exceeded 98 weeks.
In the FL subset, copanlisib produced an overall response rate of 58.7%, with a complete response rate of 14.4%. The median duration of response exceeded 52 weeks.
In the entire cohort, there were 3 deaths considered related to copanlisib.
The most common treatment-related adverse events were transient hyperglycemia (all grades: 49%/grade 3-4: 40%) and hypertension (all grades: 29%/grade 3: 23%).
“Patients with relapsed or refractory follicular lymphoma have a poor prognosis, and new treatment options which are well tolerated and effective are needed to prolong progression-free survival and improve quality of life for these patients,” said Martin Dreyling, MD, a professor at the University of Munich Hospital (Grosshadern) in Germany and lead investigator of the CHRONOS-1 study.
“Based on the CHRONOS-1 results, where copanlisib showed durable efficacy with a manageable and distinct safety profile, the compound may have the potential to address this unmet medical need.”
Data from CHRONOS-1 were presented at the AACR Annual Meeting 2017.
Data from the FL subset of the trial are scheduled to be presented at the 2017 ASCO Annual Meeting in June.
Copanlisib is being developed by Bayer. The compound has fast track and orphan drug designations from the FDA.
The FDA grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
The FDA’s fast track program is designed to facilitate the development and expedite the review of products intended to treat or prevent serious or life-threatening conditions and address unmet medical need.
Through the fast track program, a product may be eligible for priority review. In addition, the company developing the product may be allowed to submit sections of the NDA or biologic license application on a rolling basis as data become available.
Fast track designation also provides the company with opportunities for more frequent meetings and written communications with the FDA.