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Mircera approved for anemia in pediatric patients with CKD
Mircera®, methoxy polyethylene glycol-epoetin beta, was approved by the US Food and Drug Administration (FDA) to treat anemia in pediatric patients who have chronic kidney disease (CKD).
The drug is indicated for patients ages 5 to 17 years on hemodialysis who are switching from another erythropoiesis-stimulating agent (ESA) after their hemoglobin levels have stabilized.
The FDA also approved the agent to treat adult patients with CKD-associated anemia.
However, the drug is not approved to treat anemia caused by cancer chemotherapy.
The FDA based its approval on data from an open-label, multiple-dose, multicenter, dose-finding trial (NCT00717366).
Investigators enrolled 64 pediatric patients with CKD on hemodialysis. The patients had to have stable hemoglobin levels while receiving another ESA, such as epoetin alfa/beta or darbepoetin alfa.
Patients received Mircera intravenously once every 4 weeks for 20 weeks. Investigators adjusted the dosages, if necessary, after the first administration to maintain target hemoglobin levels.
Efficacy was based on the patients’ ability to maintain target hemoglobin levels and also on data extrapolated from trials of Mircera in adults with CKD.
Patients who received Mircera had a mean change in hemoglobin concentration from baseline of -0.15g/dL and 75% maintained hemoglobin values within ± 1g/dL of baseline.
Eighty-one percent maintained hemoglobin values within 10–12g/dL during the evaluation period.
The safety findings in pediatric patients were consistent with those previously reported in adults.
The most common adverse reactions occurring in 10% or more patients, as indicated in the prescribing information, are hypertension, diarrhea, and nasopharyngitis.
The drug carries a black box warning for increased risk of death, myocardial infarction, stroke, venous thromboembolism, thrombosis of vascular access, and tumor progression of recurrence.
Mircera is an erythropoietin receptor activator with greater activity in vivo as well as increased half-life, compared to erythropoietin.
Mircera is manufactured by Vifor (International) Inc.
Mircera®, methoxy polyethylene glycol-epoetin beta, was approved by the US Food and Drug Administration (FDA) to treat anemia in pediatric patients who have chronic kidney disease (CKD).
The drug is indicated for patients ages 5 to 17 years on hemodialysis who are switching from another erythropoiesis-stimulating agent (ESA) after their hemoglobin levels have stabilized.
The FDA also approved the agent to treat adult patients with CKD-associated anemia.
However, the drug is not approved to treat anemia caused by cancer chemotherapy.
The FDA based its approval on data from an open-label, multiple-dose, multicenter, dose-finding trial (NCT00717366).
Investigators enrolled 64 pediatric patients with CKD on hemodialysis. The patients had to have stable hemoglobin levels while receiving another ESA, such as epoetin alfa/beta or darbepoetin alfa.
Patients received Mircera intravenously once every 4 weeks for 20 weeks. Investigators adjusted the dosages, if necessary, after the first administration to maintain target hemoglobin levels.
Efficacy was based on the patients’ ability to maintain target hemoglobin levels and also on data extrapolated from trials of Mircera in adults with CKD.
Patients who received Mircera had a mean change in hemoglobin concentration from baseline of -0.15g/dL and 75% maintained hemoglobin values within ± 1g/dL of baseline.
Eighty-one percent maintained hemoglobin values within 10–12g/dL during the evaluation period.
The safety findings in pediatric patients were consistent with those previously reported in adults.
The most common adverse reactions occurring in 10% or more patients, as indicated in the prescribing information, are hypertension, diarrhea, and nasopharyngitis.
The drug carries a black box warning for increased risk of death, myocardial infarction, stroke, venous thromboembolism, thrombosis of vascular access, and tumor progression of recurrence.
Mircera is an erythropoietin receptor activator with greater activity in vivo as well as increased half-life, compared to erythropoietin.
Mircera is manufactured by Vifor (International) Inc.
Mircera®, methoxy polyethylene glycol-epoetin beta, was approved by the US Food and Drug Administration (FDA) to treat anemia in pediatric patients who have chronic kidney disease (CKD).
The drug is indicated for patients ages 5 to 17 years on hemodialysis who are switching from another erythropoiesis-stimulating agent (ESA) after their hemoglobin levels have stabilized.
The FDA also approved the agent to treat adult patients with CKD-associated anemia.
However, the drug is not approved to treat anemia caused by cancer chemotherapy.
The FDA based its approval on data from an open-label, multiple-dose, multicenter, dose-finding trial (NCT00717366).
Investigators enrolled 64 pediatric patients with CKD on hemodialysis. The patients had to have stable hemoglobin levels while receiving another ESA, such as epoetin alfa/beta or darbepoetin alfa.
Patients received Mircera intravenously once every 4 weeks for 20 weeks. Investigators adjusted the dosages, if necessary, after the first administration to maintain target hemoglobin levels.
Efficacy was based on the patients’ ability to maintain target hemoglobin levels and also on data extrapolated from trials of Mircera in adults with CKD.
Patients who received Mircera had a mean change in hemoglobin concentration from baseline of -0.15g/dL and 75% maintained hemoglobin values within ± 1g/dL of baseline.
Eighty-one percent maintained hemoglobin values within 10–12g/dL during the evaluation period.
The safety findings in pediatric patients were consistent with those previously reported in adults.
The most common adverse reactions occurring in 10% or more patients, as indicated in the prescribing information, are hypertension, diarrhea, and nasopharyngitis.
The drug carries a black box warning for increased risk of death, myocardial infarction, stroke, venous thromboembolism, thrombosis of vascular access, and tumor progression of recurrence.
Mircera is an erythropoietin receptor activator with greater activity in vivo as well as increased half-life, compared to erythropoietin.
Mircera is manufactured by Vifor (International) Inc.
FDA approves venetoclax for CLL/SLL with or without del 17p
The US Food and Drug Administration (FDA) has approved venetoclax tablets (Venclexta ®) in combination with rituximab to treat patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received 1 prior therapy.
The combination is approved for patients with or without deletion of 17p (del 17p).
The FDA based its approval on the phase 3 MURANO trial, in which venetoclax in combination with rituximab (VEN+R) significantly improved progression-free survival (PFS) in relapsed or refractory CLL patients compared to the chemoimmunotherapy regimen of bendamustine plus rituximab(B+R).
This approval, according to the drug’s developers, makes venetoclax plus rituximab the first oral-based, chemotherapy-free combination with a fixed treatment duration for CLL.
The FDA has also converted venetoclax's accelerated approval to a full approval. The drug was previously granted accelerated approval as a single agent for the treatment of people with CLL with 17p deletion.
Venetoclax is being developed by AbbVie and Roche and jointly commercialized by AbbVie and Genentech in the US and by AbbVie outside the US.
Phase 3 MURANO trial (NCT02005471)
The multicenter, open-label trial randomized 389 patients to VEN+R (194 patients) or B+R (195 patients). Median age of the patients was 65 years (range, 22 – 85).
Patients in the VEN+R arm completed a 5-week ramp-up of venetoclax followed by venetoclax 400 mg once daily for 24 months measured from the rituximab start date.
Tumor lysis syndrome (TLS), caused by a rapid reduction in tumor volume, is an identified risk with venetoclax treatment. The dose ramp-up was intended to mitigate this risk.
Rituximab was initiated after venetoclax ramp-up and given for 6 cycles (375 mg/m2 intravenously on cycle 1 day 1 and 500 mg/m2 intravenously on day 1 of cycles 2-6, with a 28-day cycle length).
Patients in the B+R arm received 6 cycles of B+R (bendamustine 70 mg/m2 on days 1 and 2 of each 28-day cycle and rituximab at the above described dose and schedule).
Efficacy was based on PFS as assessed by an independent review committee.
After a median follow-up of 23 months, the median PFS was not reached in the VEN+R arm and was 18.1 months in the B+R arm (P<0.0001).
The overall response rate was 92% for patients treated with VEN+R compared to 72% for those treated with B+R.
Safety
The most common adverse events (AEs) in the VEN+R arms that occurred in 20% or more patients were neutropenia (65%), diarrhea (40%), upper respiratory tract infection (39%), fatigue (22%), cough (22%), and nausea (21%).
Grade 3 or 4 neutropenia developed in 64% of patients, and grade 4 neutropenia in 31%.
Serious adverse events (SAEs) developed in 46% of patients and serious infections in 21%, consisting most frequently of pneumonia (9%).
The incidence of TLS was 3%, occurring in 6 of 194 patients.
In the VEN+R arm, discontinuations due to any AEs occurred in 16% of patients, dose reductions in 15%, and dose interruptions in 71%.
Neutropenia led to dose interruptions in 46% of patients and discontinuations in 3%. Thrombocytopenia led to discontinuations in 3% of patients.
Fatal AEs that occurred in the absence of disease progression and within 30 days of the last VEN+R treatment and/or 90 days of the last rituximab infusion were reported in 2% (4/194) of patients.
In the B+R arm, AEs led to treatment discontinuations in 10% of patients, dose reductions in 15%, and dose interruptions in 40 %.
Investigators previously reported data from the phase 3 MURANO study as a late-breaking abstract at the 2017 ASH Annual Meeting and published the findings in NEJM.
John Seymour, MBBS, PhD, lead investigator of the MURANO study, said in the corporate release, the approval "validates the results seen in the phase 3 trial, including the significant improvement in progression-free survival over a standard of care comparator arm."
"Progression-free survival is considered a gold standard for demonstrating clinical benefit in oncology," he added.
Full prescribing information for venetoclax is available here.
The US Food and Drug Administration (FDA) has approved venetoclax tablets (Venclexta ®) in combination with rituximab to treat patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received 1 prior therapy.
The combination is approved for patients with or without deletion of 17p (del 17p).
The FDA based its approval on the phase 3 MURANO trial, in which venetoclax in combination with rituximab (VEN+R) significantly improved progression-free survival (PFS) in relapsed or refractory CLL patients compared to the chemoimmunotherapy regimen of bendamustine plus rituximab(B+R).
This approval, according to the drug’s developers, makes venetoclax plus rituximab the first oral-based, chemotherapy-free combination with a fixed treatment duration for CLL.
The FDA has also converted venetoclax's accelerated approval to a full approval. The drug was previously granted accelerated approval as a single agent for the treatment of people with CLL with 17p deletion.
Venetoclax is being developed by AbbVie and Roche and jointly commercialized by AbbVie and Genentech in the US and by AbbVie outside the US.
Phase 3 MURANO trial (NCT02005471)
The multicenter, open-label trial randomized 389 patients to VEN+R (194 patients) or B+R (195 patients). Median age of the patients was 65 years (range, 22 – 85).
Patients in the VEN+R arm completed a 5-week ramp-up of venetoclax followed by venetoclax 400 mg once daily for 24 months measured from the rituximab start date.
Tumor lysis syndrome (TLS), caused by a rapid reduction in tumor volume, is an identified risk with venetoclax treatment. The dose ramp-up was intended to mitigate this risk.
Rituximab was initiated after venetoclax ramp-up and given for 6 cycles (375 mg/m2 intravenously on cycle 1 day 1 and 500 mg/m2 intravenously on day 1 of cycles 2-6, with a 28-day cycle length).
Patients in the B+R arm received 6 cycles of B+R (bendamustine 70 mg/m2 on days 1 and 2 of each 28-day cycle and rituximab at the above described dose and schedule).
Efficacy was based on PFS as assessed by an independent review committee.
After a median follow-up of 23 months, the median PFS was not reached in the VEN+R arm and was 18.1 months in the B+R arm (P<0.0001).
The overall response rate was 92% for patients treated with VEN+R compared to 72% for those treated with B+R.
Safety
The most common adverse events (AEs) in the VEN+R arms that occurred in 20% or more patients were neutropenia (65%), diarrhea (40%), upper respiratory tract infection (39%), fatigue (22%), cough (22%), and nausea (21%).
Grade 3 or 4 neutropenia developed in 64% of patients, and grade 4 neutropenia in 31%.
Serious adverse events (SAEs) developed in 46% of patients and serious infections in 21%, consisting most frequently of pneumonia (9%).
The incidence of TLS was 3%, occurring in 6 of 194 patients.
In the VEN+R arm, discontinuations due to any AEs occurred in 16% of patients, dose reductions in 15%, and dose interruptions in 71%.
Neutropenia led to dose interruptions in 46% of patients and discontinuations in 3%. Thrombocytopenia led to discontinuations in 3% of patients.
Fatal AEs that occurred in the absence of disease progression and within 30 days of the last VEN+R treatment and/or 90 days of the last rituximab infusion were reported in 2% (4/194) of patients.
In the B+R arm, AEs led to treatment discontinuations in 10% of patients, dose reductions in 15%, and dose interruptions in 40 %.
Investigators previously reported data from the phase 3 MURANO study as a late-breaking abstract at the 2017 ASH Annual Meeting and published the findings in NEJM.
John Seymour, MBBS, PhD, lead investigator of the MURANO study, said in the corporate release, the approval "validates the results seen in the phase 3 trial, including the significant improvement in progression-free survival over a standard of care comparator arm."
"Progression-free survival is considered a gold standard for demonstrating clinical benefit in oncology," he added.
Full prescribing information for venetoclax is available here.
The US Food and Drug Administration (FDA) has approved venetoclax tablets (Venclexta ®) in combination with rituximab to treat patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received 1 prior therapy.
The combination is approved for patients with or without deletion of 17p (del 17p).
The FDA based its approval on the phase 3 MURANO trial, in which venetoclax in combination with rituximab (VEN+R) significantly improved progression-free survival (PFS) in relapsed or refractory CLL patients compared to the chemoimmunotherapy regimen of bendamustine plus rituximab(B+R).
This approval, according to the drug’s developers, makes venetoclax plus rituximab the first oral-based, chemotherapy-free combination with a fixed treatment duration for CLL.
The FDA has also converted venetoclax's accelerated approval to a full approval. The drug was previously granted accelerated approval as a single agent for the treatment of people with CLL with 17p deletion.
Venetoclax is being developed by AbbVie and Roche and jointly commercialized by AbbVie and Genentech in the US and by AbbVie outside the US.
Phase 3 MURANO trial (NCT02005471)
The multicenter, open-label trial randomized 389 patients to VEN+R (194 patients) or B+R (195 patients). Median age of the patients was 65 years (range, 22 – 85).
Patients in the VEN+R arm completed a 5-week ramp-up of venetoclax followed by venetoclax 400 mg once daily for 24 months measured from the rituximab start date.
Tumor lysis syndrome (TLS), caused by a rapid reduction in tumor volume, is an identified risk with venetoclax treatment. The dose ramp-up was intended to mitigate this risk.
Rituximab was initiated after venetoclax ramp-up and given for 6 cycles (375 mg/m2 intravenously on cycle 1 day 1 and 500 mg/m2 intravenously on day 1 of cycles 2-6, with a 28-day cycle length).
Patients in the B+R arm received 6 cycles of B+R (bendamustine 70 mg/m2 on days 1 and 2 of each 28-day cycle and rituximab at the above described dose and schedule).
Efficacy was based on PFS as assessed by an independent review committee.
After a median follow-up of 23 months, the median PFS was not reached in the VEN+R arm and was 18.1 months in the B+R arm (P<0.0001).
The overall response rate was 92% for patients treated with VEN+R compared to 72% for those treated with B+R.
Safety
The most common adverse events (AEs) in the VEN+R arms that occurred in 20% or more patients were neutropenia (65%), diarrhea (40%), upper respiratory tract infection (39%), fatigue (22%), cough (22%), and nausea (21%).
Grade 3 or 4 neutropenia developed in 64% of patients, and grade 4 neutropenia in 31%.
Serious adverse events (SAEs) developed in 46% of patients and serious infections in 21%, consisting most frequently of pneumonia (9%).
The incidence of TLS was 3%, occurring in 6 of 194 patients.
In the VEN+R arm, discontinuations due to any AEs occurred in 16% of patients, dose reductions in 15%, and dose interruptions in 71%.
Neutropenia led to dose interruptions in 46% of patients and discontinuations in 3%. Thrombocytopenia led to discontinuations in 3% of patients.
Fatal AEs that occurred in the absence of disease progression and within 30 days of the last VEN+R treatment and/or 90 days of the last rituximab infusion were reported in 2% (4/194) of patients.
In the B+R arm, AEs led to treatment discontinuations in 10% of patients, dose reductions in 15%, and dose interruptions in 40 %.
Investigators previously reported data from the phase 3 MURANO study as a late-breaking abstract at the 2017 ASH Annual Meeting and published the findings in NEJM.
John Seymour, MBBS, PhD, lead investigator of the MURANO study, said in the corporate release, the approval "validates the results seen in the phase 3 trial, including the significant improvement in progression-free survival over a standard of care comparator arm."
"Progression-free survival is considered a gold standard for demonstrating clinical benefit in oncology," he added.
Full prescribing information for venetoclax is available here.
Emicizumab granted priority review for hemophilia A without inhibitors
The US Food and Drug Administration (FDA) has granted priority review for emicizumab (Hemlibra®) for adults and children with hemophilia A without factor VIII inhibitors.
Earlier this year, the agency awarded emicizumab breakthrough therapy designation for the same population.
Emicizumab is a bispecific factor IXa- and factor X-directed antibody approved by the FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children who have hemophilia A with factor VIII inhibitors.
The FDA based its decision to grant emicizumab priority review on the phase 3 HAVEN 3 study, results of which were presented recently at the World Federation of Hemophilia congress.
In HAVEN 3, emicizumab demonstrated a 68% reduction (P<0.0001) in treated bleeds based on an intra-patient comparison in patients who were previously enrolled in a prospective non-interventional study.
According to Genentech, co-developer of the drug, this makes emicizumab the first medicine to show superior efficacy to prior treatment with factor VIII prophylaxis, the current standard of care for people with hemophilia A without factor VIII inhibitors.
About HAVEN 3
The randomized, multicenter, open-label trial evaluated prophylaxis versus no prophylaxis in patients without factor VIII inhibitors.
The study included 152 patients 12 years or older who were previously treated with factor VIII therapy on-demand or as prophylaxis.
Patients previously treated with on-demand factor VIII were randomized in a 2:2:1 fashion to 1 of 3 treatment groups:
- Arm A received emicizumab prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study.
- Arm B received emicizumab prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 3 mg/kg/2wks for at least 24 weeks.
- Arm C received no prophylaxis
Patients previously treated prophylactically with factor VIII were enrolled in Arm D and received emicizumab prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study.
The protocol permitted episodic treatment of breakthrough bleeds with factor VIII therapy.
Patients in the prophylaxis groups achieved a 96% (P<0.0001) and 97% (P<0.0001) reduction in treated bleeds, respectively, compared to those who received no prophylaxis.
Additionally, 55.6% of patients treated weekly and 60% treated every 2 weeks had no treated bleeds. In contrast, 0% in the prophylaxis group achieved zero treated bleeds.
Investigators observed no unexpected or serious adverse events (AEs), no thrombotic events, and no cases of thrombotic microangiopathy.
The most common AEs occurring in 5% or more of patients were injection site reactions, arthralgia, nasopharyngitis, headache, upper respiratory tract infection, and influenza.
The FDA is expected to make a decision regarding approval by October 4.
The US Food and Drug Administration (FDA) has granted priority review for emicizumab (Hemlibra®) for adults and children with hemophilia A without factor VIII inhibitors.
Earlier this year, the agency awarded emicizumab breakthrough therapy designation for the same population.
Emicizumab is a bispecific factor IXa- and factor X-directed antibody approved by the FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children who have hemophilia A with factor VIII inhibitors.
The FDA based its decision to grant emicizumab priority review on the phase 3 HAVEN 3 study, results of which were presented recently at the World Federation of Hemophilia congress.
In HAVEN 3, emicizumab demonstrated a 68% reduction (P<0.0001) in treated bleeds based on an intra-patient comparison in patients who were previously enrolled in a prospective non-interventional study.
According to Genentech, co-developer of the drug, this makes emicizumab the first medicine to show superior efficacy to prior treatment with factor VIII prophylaxis, the current standard of care for people with hemophilia A without factor VIII inhibitors.
About HAVEN 3
The randomized, multicenter, open-label trial evaluated prophylaxis versus no prophylaxis in patients without factor VIII inhibitors.
The study included 152 patients 12 years or older who were previously treated with factor VIII therapy on-demand or as prophylaxis.
Patients previously treated with on-demand factor VIII were randomized in a 2:2:1 fashion to 1 of 3 treatment groups:
- Arm A received emicizumab prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study.
- Arm B received emicizumab prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 3 mg/kg/2wks for at least 24 weeks.
- Arm C received no prophylaxis
Patients previously treated prophylactically with factor VIII were enrolled in Arm D and received emicizumab prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study.
The protocol permitted episodic treatment of breakthrough bleeds with factor VIII therapy.
Patients in the prophylaxis groups achieved a 96% (P<0.0001) and 97% (P<0.0001) reduction in treated bleeds, respectively, compared to those who received no prophylaxis.
Additionally, 55.6% of patients treated weekly and 60% treated every 2 weeks had no treated bleeds. In contrast, 0% in the prophylaxis group achieved zero treated bleeds.
Investigators observed no unexpected or serious adverse events (AEs), no thrombotic events, and no cases of thrombotic microangiopathy.
The most common AEs occurring in 5% or more of patients were injection site reactions, arthralgia, nasopharyngitis, headache, upper respiratory tract infection, and influenza.
The FDA is expected to make a decision regarding approval by October 4.
The US Food and Drug Administration (FDA) has granted priority review for emicizumab (Hemlibra®) for adults and children with hemophilia A without factor VIII inhibitors.
Earlier this year, the agency awarded emicizumab breakthrough therapy designation for the same population.
Emicizumab is a bispecific factor IXa- and factor X-directed antibody approved by the FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children who have hemophilia A with factor VIII inhibitors.
The FDA based its decision to grant emicizumab priority review on the phase 3 HAVEN 3 study, results of which were presented recently at the World Federation of Hemophilia congress.
In HAVEN 3, emicizumab demonstrated a 68% reduction (P<0.0001) in treated bleeds based on an intra-patient comparison in patients who were previously enrolled in a prospective non-interventional study.
According to Genentech, co-developer of the drug, this makes emicizumab the first medicine to show superior efficacy to prior treatment with factor VIII prophylaxis, the current standard of care for people with hemophilia A without factor VIII inhibitors.
About HAVEN 3
The randomized, multicenter, open-label trial evaluated prophylaxis versus no prophylaxis in patients without factor VIII inhibitors.
The study included 152 patients 12 years or older who were previously treated with factor VIII therapy on-demand or as prophylaxis.
Patients previously treated with on-demand factor VIII were randomized in a 2:2:1 fashion to 1 of 3 treatment groups:
- Arm A received emicizumab prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study.
- Arm B received emicizumab prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 3 mg/kg/2wks for at least 24 weeks.
- Arm C received no prophylaxis
Patients previously treated prophylactically with factor VIII were enrolled in Arm D and received emicizumab prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study.
The protocol permitted episodic treatment of breakthrough bleeds with factor VIII therapy.
Patients in the prophylaxis groups achieved a 96% (P<0.0001) and 97% (P<0.0001) reduction in treated bleeds, respectively, compared to those who received no prophylaxis.
Additionally, 55.6% of patients treated weekly and 60% treated every 2 weeks had no treated bleeds. In contrast, 0% in the prophylaxis group achieved zero treated bleeds.
Investigators observed no unexpected or serious adverse events (AEs), no thrombotic events, and no cases of thrombotic microangiopathy.
The most common AEs occurring in 5% or more of patients were injection site reactions, arthralgia, nasopharyngitis, headache, upper respiratory tract infection, and influenza.
The FDA is expected to make a decision regarding approval by October 4.
FDA approves first biosimilar pegfilgrastim
The US Food and Drug Association (FDA) has approved pegfilgrastim-jmdb (Fulphila™) as the first biosimilar to Neulasta®.
The agents reduce the risk of infection or the duration of febrile neutropenia in patients treated with immunosuppressive chemotherapy for non-myeloid hematologic malignancies.
The FDA approved Fulphila based on evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamic data, clinical immunogenicity data, and other clinical safety and effectiveness data.
The evidence demonstrated that Fulphila is biosimilar to Amgen’s Neulasta. The FDA, in its announcement, noted that Fulphila has been approved as a biosimilar and not as an interchangeable product.
A biosimilar is a biological product approved based on data showing it is highly similar to a biological product already approved by the FDA, termed the reference product.
A biosimilar has no clinically meaningful differences from the reference product in terms of safety, purity, and effectiveness.
Common side effects of Fulphila include bone pain and pain in extremities.
The FDA cautions that patients with a history of serious allergic reaction to human granulocyte colony-stimulating factors, such as pegfilgrastim or filgrastim products, should not take Fulphila.
Serious side effects from Fulphila include:
- rupture of the spleen
- acute respiratory distress syndrome
- serious allergic reactions including anaphylaxis
- glomerulonephritis
- leukocytosis
- capillary leak syndrome
- potential for tumor growth
Fatal sickle cell crises have also occurred with Fulphila use.
Fulphila is not indicated for the mobilization of peripheral blood progenitor cells for hematopoietic stem cell transplantation.
The FDA is planning to release a comprehensive new plan to advance policy efforts that promote biosimilar product development, according to FDA Commissioner Scott Gotlieb, MD.
“We want to make sure that the pathway for developing biosimilar versions of approved biologics is efficient and effective, so that patients benefit from competition to existing biologics once lawful intellectual property has lapsed on these products,” he said in the announcement.
The FDA granted approval of Fulphila to Mylan GmbH. Mylan is co-developing Fulphila with Biocon.
Last fall, the agency had issued a complete response letter saying it could not approve the proposed biosimilar pending an update to the application.
The complete response letter did not raise any questions on the biosimilarity of Fulphila (investigational drug product MYL-1401H), pharmacokinetic/pharmacodynamic data, clinical data, or immunogenicity, however.
Mylan anticipates launching Fulphila in the coming weeks.
The US Food and Drug Association (FDA) has approved pegfilgrastim-jmdb (Fulphila™) as the first biosimilar to Neulasta®.
The agents reduce the risk of infection or the duration of febrile neutropenia in patients treated with immunosuppressive chemotherapy for non-myeloid hematologic malignancies.
The FDA approved Fulphila based on evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamic data, clinical immunogenicity data, and other clinical safety and effectiveness data.
The evidence demonstrated that Fulphila is biosimilar to Amgen’s Neulasta. The FDA, in its announcement, noted that Fulphila has been approved as a biosimilar and not as an interchangeable product.
A biosimilar is a biological product approved based on data showing it is highly similar to a biological product already approved by the FDA, termed the reference product.
A biosimilar has no clinically meaningful differences from the reference product in terms of safety, purity, and effectiveness.
Common side effects of Fulphila include bone pain and pain in extremities.
The FDA cautions that patients with a history of serious allergic reaction to human granulocyte colony-stimulating factors, such as pegfilgrastim or filgrastim products, should not take Fulphila.
Serious side effects from Fulphila include:
- rupture of the spleen
- acute respiratory distress syndrome
- serious allergic reactions including anaphylaxis
- glomerulonephritis
- leukocytosis
- capillary leak syndrome
- potential for tumor growth
Fatal sickle cell crises have also occurred with Fulphila use.
Fulphila is not indicated for the mobilization of peripheral blood progenitor cells for hematopoietic stem cell transplantation.
The FDA is planning to release a comprehensive new plan to advance policy efforts that promote biosimilar product development, according to FDA Commissioner Scott Gotlieb, MD.
“We want to make sure that the pathway for developing biosimilar versions of approved biologics is efficient and effective, so that patients benefit from competition to existing biologics once lawful intellectual property has lapsed on these products,” he said in the announcement.
The FDA granted approval of Fulphila to Mylan GmbH. Mylan is co-developing Fulphila with Biocon.
Last fall, the agency had issued a complete response letter saying it could not approve the proposed biosimilar pending an update to the application.
The complete response letter did not raise any questions on the biosimilarity of Fulphila (investigational drug product MYL-1401H), pharmacokinetic/pharmacodynamic data, clinical data, or immunogenicity, however.
Mylan anticipates launching Fulphila in the coming weeks.
The US Food and Drug Association (FDA) has approved pegfilgrastim-jmdb (Fulphila™) as the first biosimilar to Neulasta®.
The agents reduce the risk of infection or the duration of febrile neutropenia in patients treated with immunosuppressive chemotherapy for non-myeloid hematologic malignancies.
The FDA approved Fulphila based on evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamic data, clinical immunogenicity data, and other clinical safety and effectiveness data.
The evidence demonstrated that Fulphila is biosimilar to Amgen’s Neulasta. The FDA, in its announcement, noted that Fulphila has been approved as a biosimilar and not as an interchangeable product.
A biosimilar is a biological product approved based on data showing it is highly similar to a biological product already approved by the FDA, termed the reference product.
A biosimilar has no clinically meaningful differences from the reference product in terms of safety, purity, and effectiveness.
Common side effects of Fulphila include bone pain and pain in extremities.
The FDA cautions that patients with a history of serious allergic reaction to human granulocyte colony-stimulating factors, such as pegfilgrastim or filgrastim products, should not take Fulphila.
Serious side effects from Fulphila include:
- rupture of the spleen
- acute respiratory distress syndrome
- serious allergic reactions including anaphylaxis
- glomerulonephritis
- leukocytosis
- capillary leak syndrome
- potential for tumor growth
Fatal sickle cell crises have also occurred with Fulphila use.
Fulphila is not indicated for the mobilization of peripheral blood progenitor cells for hematopoietic stem cell transplantation.
The FDA is planning to release a comprehensive new plan to advance policy efforts that promote biosimilar product development, according to FDA Commissioner Scott Gotlieb, MD.
“We want to make sure that the pathway for developing biosimilar versions of approved biologics is efficient and effective, so that patients benefit from competition to existing biologics once lawful intellectual property has lapsed on these products,” he said in the announcement.
The FDA granted approval of Fulphila to Mylan GmbH. Mylan is co-developing Fulphila with Biocon.
Last fall, the agency had issued a complete response letter saying it could not approve the proposed biosimilar pending an update to the application.
The complete response letter did not raise any questions on the biosimilarity of Fulphila (investigational drug product MYL-1401H), pharmacokinetic/pharmacodynamic data, clinical data, or immunogenicity, however.
Mylan anticipates launching Fulphila in the coming weeks.
Over 1100 new meds, vaccines being developed to treat cancer
Currently, 1,120 new medicines and vaccines are being developed to treat cancer, according to a new report of the Pharmaceutical Research and Manufacturers of America (PhRMA).
And all of them, the organization states, are in clinical trials or awaiting review by the US Food and Drug Administration (FDA).
Leading the way are treatments for solid tumors, with 397 in development. Treatments for blood cancers are not far behind, with nearly 340 medicines in development: 137 for leukemias, 135 for lymphomas, and 62 for multiple myeloma.
Immuno-oncology and personalized medicine have a hand in this increase.
In the last year, according to PhRMA’s "Medicines in Development for Cancer 2018 Report," 47 new immune-oncology treatments have been added to the development pipeline, including CAR-T therapies and checkpoint inhibitors.
This brings the total to 295 immuno-oncology medicines and vaccines in the development pipeline this year.
The report also states that about 85% of these medicines in the oncology pipeline are first-in-class.
And PhRMA attributes the approximately 73% of survival gains in cancer to the new medicines.
Despite the bright picture, PhRMA acknowledges the financial burden and medical care challenges patients encounter.
It addresses them in a new chart pack, "Cancer Medicines: Value in Context," which puts cancer costs in perspective and offers solutions for improving the current system in the United States.
The association reports the top medical financial concerns of patients to be diagnostic tests or scans (53%), prescription medicines (43%), physician office visits (39%), outpatient treatments-including radiation (37%), and surgery (36%).
Spending on cancer medicines represents about 1% of overall healthcare spending, according to the organization, with cancer medications accounting for $49.8 billion of the $3.49 trillion healthcare spending in the United States.
Cancer medicines represent about 20% of spending on cancer, PhrMA notes, and some insurance plans place treatments for certain high-cost conditions on the highest drug formulary cost-sharing tier.
And patients with the highest copay were 5 times more likely to abandon treatment than the lowest copay group, PhRMA points out.
“No patient should struggle to afford their needed treatments,” PhRMA stated in a release, “and it is important that we address patient access challenges.”
Currently, 1,120 new medicines and vaccines are being developed to treat cancer, according to a new report of the Pharmaceutical Research and Manufacturers of America (PhRMA).
And all of them, the organization states, are in clinical trials or awaiting review by the US Food and Drug Administration (FDA).
Leading the way are treatments for solid tumors, with 397 in development. Treatments for blood cancers are not far behind, with nearly 340 medicines in development: 137 for leukemias, 135 for lymphomas, and 62 for multiple myeloma.
Immuno-oncology and personalized medicine have a hand in this increase.
In the last year, according to PhRMA’s "Medicines in Development for Cancer 2018 Report," 47 new immune-oncology treatments have been added to the development pipeline, including CAR-T therapies and checkpoint inhibitors.
This brings the total to 295 immuno-oncology medicines and vaccines in the development pipeline this year.
The report also states that about 85% of these medicines in the oncology pipeline are first-in-class.
And PhRMA attributes the approximately 73% of survival gains in cancer to the new medicines.
Despite the bright picture, PhRMA acknowledges the financial burden and medical care challenges patients encounter.
It addresses them in a new chart pack, "Cancer Medicines: Value in Context," which puts cancer costs in perspective and offers solutions for improving the current system in the United States.
The association reports the top medical financial concerns of patients to be diagnostic tests or scans (53%), prescription medicines (43%), physician office visits (39%), outpatient treatments-including radiation (37%), and surgery (36%).
Spending on cancer medicines represents about 1% of overall healthcare spending, according to the organization, with cancer medications accounting for $49.8 billion of the $3.49 trillion healthcare spending in the United States.
Cancer medicines represent about 20% of spending on cancer, PhrMA notes, and some insurance plans place treatments for certain high-cost conditions on the highest drug formulary cost-sharing tier.
And patients with the highest copay were 5 times more likely to abandon treatment than the lowest copay group, PhRMA points out.
“No patient should struggle to afford their needed treatments,” PhRMA stated in a release, “and it is important that we address patient access challenges.”
Currently, 1,120 new medicines and vaccines are being developed to treat cancer, according to a new report of the Pharmaceutical Research and Manufacturers of America (PhRMA).
And all of them, the organization states, are in clinical trials or awaiting review by the US Food and Drug Administration (FDA).
Leading the way are treatments for solid tumors, with 397 in development. Treatments for blood cancers are not far behind, with nearly 340 medicines in development: 137 for leukemias, 135 for lymphomas, and 62 for multiple myeloma.
Immuno-oncology and personalized medicine have a hand in this increase.
In the last year, according to PhRMA’s "Medicines in Development for Cancer 2018 Report," 47 new immune-oncology treatments have been added to the development pipeline, including CAR-T therapies and checkpoint inhibitors.
This brings the total to 295 immuno-oncology medicines and vaccines in the development pipeline this year.
The report also states that about 85% of these medicines in the oncology pipeline are first-in-class.
And PhRMA attributes the approximately 73% of survival gains in cancer to the new medicines.
Despite the bright picture, PhRMA acknowledges the financial burden and medical care challenges patients encounter.
It addresses them in a new chart pack, "Cancer Medicines: Value in Context," which puts cancer costs in perspective and offers solutions for improving the current system in the United States.
The association reports the top medical financial concerns of patients to be diagnostic tests or scans (53%), prescription medicines (43%), physician office visits (39%), outpatient treatments-including radiation (37%), and surgery (36%).
Spending on cancer medicines represents about 1% of overall healthcare spending, according to the organization, with cancer medications accounting for $49.8 billion of the $3.49 trillion healthcare spending in the United States.
Cancer medicines represent about 20% of spending on cancer, PhrMA notes, and some insurance plans place treatments for certain high-cost conditions on the highest drug formulary cost-sharing tier.
And patients with the highest copay were 5 times more likely to abandon treatment than the lowest copay group, PhRMA points out.
“No patient should struggle to afford their needed treatments,” PhRMA stated in a release, “and it is important that we address patient access challenges.”
Eltrombopag receives priority review designation for SAA
Eltrombopag (Promacta®) in combination with standard immunosuppressive therapy (IST) has received priority review designation from the US Food and Drug Administration (FDA) for first-line treatment of severe aplastic anemia (SAA).
The drug is already approved for SAA in the refractory setting for patients who have had an insufficient response to IST.
And it is approved for adults and children with chronic immune thrombocytopenia (ITP) who are refractory to other treatments and for patients with chronic hepatitis C virus infection who are thrombocytopenic.
Eltrombopag, an oral thrombopoietin receptor agonist, had received breakthrough therapy designation from the FDA earlier this year for use in combination with IST as first-line treatment of SAA.
The priority review designation for the agent as a first-line treatment for SAA is supported by data from a phase 1/2 trial published in NEJM in April 2017 and subsequent data on file with Novartis.
The FDA intends to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The agency grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
Trial data
Ninety-two patients with previously untreated SAA were enrolled on the trial and received IST and eltrombopag in 3 different cohorts.
Cohorts varied by start day of eltrombopag and duration of eltrombopag therapy. Patients in cohort 1 received eltrombopag from day 14 to 6 months. Patients in cohort 2 received the drug from day 14 to 3 months. And patients in cohort 3 received eltrombopag from day 1 to 6 months.
The overall response rate (ORR) at 6 months was 80% (cohort 1), 87% (cohort 2), and 94% (cohort 3).
The complete response rate at 6 months was 33%, 26%, and 58% in the 3 cohorts, respectively.
At a median follow-up of 2 years, the overall survival rate was 97%.
In the corporate announcement of the priority review designation, Novartis reported an ORR of 85% at 6 months.
Adverse events included transient elevations in liver enzyme levels (7 patients) and 2 severe adverse events—grades 2 and 3 cutaneous eruption—related to eltrombopag that resulted in patients stopping the drug.
Eltrombopag is marketed as Revolade in countries outside the US.
Eltrombopag (Promacta®) in combination with standard immunosuppressive therapy (IST) has received priority review designation from the US Food and Drug Administration (FDA) for first-line treatment of severe aplastic anemia (SAA).
The drug is already approved for SAA in the refractory setting for patients who have had an insufficient response to IST.
And it is approved for adults and children with chronic immune thrombocytopenia (ITP) who are refractory to other treatments and for patients with chronic hepatitis C virus infection who are thrombocytopenic.
Eltrombopag, an oral thrombopoietin receptor agonist, had received breakthrough therapy designation from the FDA earlier this year for use in combination with IST as first-line treatment of SAA.
The priority review designation for the agent as a first-line treatment for SAA is supported by data from a phase 1/2 trial published in NEJM in April 2017 and subsequent data on file with Novartis.
The FDA intends to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The agency grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
Trial data
Ninety-two patients with previously untreated SAA were enrolled on the trial and received IST and eltrombopag in 3 different cohorts.
Cohorts varied by start day of eltrombopag and duration of eltrombopag therapy. Patients in cohort 1 received eltrombopag from day 14 to 6 months. Patients in cohort 2 received the drug from day 14 to 3 months. And patients in cohort 3 received eltrombopag from day 1 to 6 months.
The overall response rate (ORR) at 6 months was 80% (cohort 1), 87% (cohort 2), and 94% (cohort 3).
The complete response rate at 6 months was 33%, 26%, and 58% in the 3 cohorts, respectively.
At a median follow-up of 2 years, the overall survival rate was 97%.
In the corporate announcement of the priority review designation, Novartis reported an ORR of 85% at 6 months.
Adverse events included transient elevations in liver enzyme levels (7 patients) and 2 severe adverse events—grades 2 and 3 cutaneous eruption—related to eltrombopag that resulted in patients stopping the drug.
Eltrombopag is marketed as Revolade in countries outside the US.
Eltrombopag (Promacta®) in combination with standard immunosuppressive therapy (IST) has received priority review designation from the US Food and Drug Administration (FDA) for first-line treatment of severe aplastic anemia (SAA).
The drug is already approved for SAA in the refractory setting for patients who have had an insufficient response to IST.
And it is approved for adults and children with chronic immune thrombocytopenia (ITP) who are refractory to other treatments and for patients with chronic hepatitis C virus infection who are thrombocytopenic.
Eltrombopag, an oral thrombopoietin receptor agonist, had received breakthrough therapy designation from the FDA earlier this year for use in combination with IST as first-line treatment of SAA.
The priority review designation for the agent as a first-line treatment for SAA is supported by data from a phase 1/2 trial published in NEJM in April 2017 and subsequent data on file with Novartis.
The FDA intends to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The agency grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
Trial data
Ninety-two patients with previously untreated SAA were enrolled on the trial and received IST and eltrombopag in 3 different cohorts.
Cohorts varied by start day of eltrombopag and duration of eltrombopag therapy. Patients in cohort 1 received eltrombopag from day 14 to 6 months. Patients in cohort 2 received the drug from day 14 to 3 months. And patients in cohort 3 received eltrombopag from day 1 to 6 months.
The overall response rate (ORR) at 6 months was 80% (cohort 1), 87% (cohort 2), and 94% (cohort 3).
The complete response rate at 6 months was 33%, 26%, and 58% in the 3 cohorts, respectively.
At a median follow-up of 2 years, the overall survival rate was 97%.
In the corporate announcement of the priority review designation, Novartis reported an ORR of 85% at 6 months.
Adverse events included transient elevations in liver enzyme levels (7 patients) and 2 severe adverse events—grades 2 and 3 cutaneous eruption—related to eltrombopag that resulted in patients stopping the drug.
Eltrombopag is marketed as Revolade in countries outside the US.
FDA grants priority review to gilteritinib for R/R AML
The US Food and Drug Administration (FDA) has granted priority review to gilteritinib for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) who have a FLT3 mutation.
At present, no FLT3-targeting agents are approved for the treatment of R/R FLT3-mutation-positive AML, according to Astellas, the developer of the drug.
The FDA previously granted gilteritinib orphan drug designation and fast track designation.
The European Commission (EC) also granted gilteritinib orphan designation and the Japan Ministry of Health, Labor and Welfare (MHLW) did likewise.
Gilteritinib has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) and FLT tyrosine kinase domain (TKD) mutation. These 2 FLT3 mutations are present in approximately 1/3 of AML patients.
The priority review for the new drug application is based on the ongoing phase 3 ADMIRAL trial.
The FDA aims to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The agency grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The goal date for a decision by the FDA is the end of November.
This open-label multicenter randomized study compares gilteritinib with salvage chemotherapy in adult patients with FLT3 mutations who are refractory to or have relapsed after first-line AML therapy.
The study has enrolled 371 patients with FLT3 mutations present in bone marrow or whole blood, as determined by a central lab.
Patients were randomized in a 2:1 ratio to receive gilteritinib (120 mg) or salvage chemotherapy.
Salvage chemotherapy could consist of low-dose cytarabine, azacitidine, MEC (mitoxantrone, etoposide, cytarabine) induction chemotherapy, or FLAG-IDA (fludarabine, cytarabine, and granulocyte colony-stimulating factor [G-CSF] with idarubicin) induction chemotherapy.
The primary endpoints of the study are overall survival and complete remission or complete remission with partial hematologic recovery.
Secondary endpoints include event-free survival, complete remission rate, leukemia-free survival, duration of remission, transplantation rate, fatigue inventory, among other outcomes.
The study is estimated to be completed in October for its primary endpoint and February 2020 for the entire study.
The US Food and Drug Administration (FDA) has granted priority review to gilteritinib for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) who have a FLT3 mutation.
At present, no FLT3-targeting agents are approved for the treatment of R/R FLT3-mutation-positive AML, according to Astellas, the developer of the drug.
The FDA previously granted gilteritinib orphan drug designation and fast track designation.
The European Commission (EC) also granted gilteritinib orphan designation and the Japan Ministry of Health, Labor and Welfare (MHLW) did likewise.
Gilteritinib has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) and FLT tyrosine kinase domain (TKD) mutation. These 2 FLT3 mutations are present in approximately 1/3 of AML patients.
The priority review for the new drug application is based on the ongoing phase 3 ADMIRAL trial.
The FDA aims to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The agency grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The goal date for a decision by the FDA is the end of November.
This open-label multicenter randomized study compares gilteritinib with salvage chemotherapy in adult patients with FLT3 mutations who are refractory to or have relapsed after first-line AML therapy.
The study has enrolled 371 patients with FLT3 mutations present in bone marrow or whole blood, as determined by a central lab.
Patients were randomized in a 2:1 ratio to receive gilteritinib (120 mg) or salvage chemotherapy.
Salvage chemotherapy could consist of low-dose cytarabine, azacitidine, MEC (mitoxantrone, etoposide, cytarabine) induction chemotherapy, or FLAG-IDA (fludarabine, cytarabine, and granulocyte colony-stimulating factor [G-CSF] with idarubicin) induction chemotherapy.
The primary endpoints of the study are overall survival and complete remission or complete remission with partial hematologic recovery.
Secondary endpoints include event-free survival, complete remission rate, leukemia-free survival, duration of remission, transplantation rate, fatigue inventory, among other outcomes.
The study is estimated to be completed in October for its primary endpoint and February 2020 for the entire study.
The US Food and Drug Administration (FDA) has granted priority review to gilteritinib for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) who have a FLT3 mutation.
At present, no FLT3-targeting agents are approved for the treatment of R/R FLT3-mutation-positive AML, according to Astellas, the developer of the drug.
The FDA previously granted gilteritinib orphan drug designation and fast track designation.
The European Commission (EC) also granted gilteritinib orphan designation and the Japan Ministry of Health, Labor and Welfare (MHLW) did likewise.
Gilteritinib has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) and FLT tyrosine kinase domain (TKD) mutation. These 2 FLT3 mutations are present in approximately 1/3 of AML patients.
The priority review for the new drug application is based on the ongoing phase 3 ADMIRAL trial.
The FDA aims to take action on a priority review application within 6 months of receiving it, rather than the standard 10 months.
The agency grants priority review to applications for products that may provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The goal date for a decision by the FDA is the end of November.
This open-label multicenter randomized study compares gilteritinib with salvage chemotherapy in adult patients with FLT3 mutations who are refractory to or have relapsed after first-line AML therapy.
The study has enrolled 371 patients with FLT3 mutations present in bone marrow or whole blood, as determined by a central lab.
Patients were randomized in a 2:1 ratio to receive gilteritinib (120 mg) or salvage chemotherapy.
Salvage chemotherapy could consist of low-dose cytarabine, azacitidine, MEC (mitoxantrone, etoposide, cytarabine) induction chemotherapy, or FLAG-IDA (fludarabine, cytarabine, and granulocyte colony-stimulating factor [G-CSF] with idarubicin) induction chemotherapy.
The primary endpoints of the study are overall survival and complete remission or complete remission with partial hematologic recovery.
Secondary endpoints include event-free survival, complete remission rate, leukemia-free survival, duration of remission, transplantation rate, fatigue inventory, among other outcomes.
The study is estimated to be completed in October for its primary endpoint and February 2020 for the entire study.
Drug makers may be blocking generic competition
The US Food and Drug Administration (FDA) has released a list of companies that “may be pursuing gaming tactics to delay generic competition.”
FDA Commissioner Scott Gottlieb, MD, said the agency is taking steps to improve access to drugs and make them more affordable.
One of these steps is trying to prevent companies that market brand-name drugs from delaying the release of generic equivalents.
Dr Gottlieb explained that brand companies may create obstacles for generic developers to purchase samples of a brand-name drug.
“In general, generic drug developers need the samples of the brand drug to develop their generic product and/or to conduct testing to show that their product is bioequivalent to the brand drug for FDA approval,” he said.
“Without these samples, generic drug makers may not be able to develop generic alternatives. Yet the FDA has heard that some brand companies will adopt tactics to make it hard for the generic companies to purchase these brand drugs at a fair value and in the open marketplace.”
One such tactic is for brand companies to place restrictions in their commercial contracts or agreements with prescription drug distributors, wholesalers, or specialty pharmacies that limit their ability to sell samples to generic drug developers.
In other cases, brand companies may be preventing generic drug developers from accessing samples due to limited distribution programs required by a Risk Evaluation and Mitigation Strategy (REMS) with Elements to Assure Safe Use (ETASU).
“But I want to be very clear: a path to securing samples of brand drugs for the purpose of generic drug development should always be available,” Dr Gottlieb said. “Even in the case of limited distribution programs, such as those required by certain REMS, there should be a path forward for generic drug development.”
With this in mind, the FDA has released a list of brand companies that may have prevented generic drug developers from accessing samples. The list contains 52 brand-name drugs, including Pomalyst, Promacta, Revlimid, Tasigna, and Thalidomid.
Generic drug developers have reported trouble obtaining samples of these drugs and asked the FDA for assistance.
When the FDA receives such a request, the agency first determines whether the product in question has a REMS program with ETASU that may impact distribution.
If the FDA confirms the existence of such a program, the generic company can submit its bioequivalence testing protocol to the FDA. The agency then evaluates this protocol to ensure the plan for testing the product contains safety protections comparable to the brand product’s REMS program.
If a generic developer’s plans include appropriate protections, the developer can request that the FDA send a Safety Determination Letter to the brand company. This letter notes that the REMS program doesn’t prevent the brand company from selling its product to generic drug developers.
The FDA has issued 21 Safety Determination Letters to date, according to Dr Gottlieb.
If a generic drug developer has trouble obtaining samples of a drug for which there is no REMS program with ETASU, the FDA informs the developer that there are no FDA-required restrictions that would prevent it from obtaining samples.
The FDA also notifies the Federal Trade Commission (FTC) of requests from generic drug developers seeking assistance in obtaining samples from brand companies. And the FDA encourages generic drug developers to raise these cases with the FTC if they believe that anticompetitive conduct has taken place.
The US Food and Drug Administration (FDA) has released a list of companies that “may be pursuing gaming tactics to delay generic competition.”
FDA Commissioner Scott Gottlieb, MD, said the agency is taking steps to improve access to drugs and make them more affordable.
One of these steps is trying to prevent companies that market brand-name drugs from delaying the release of generic equivalents.
Dr Gottlieb explained that brand companies may create obstacles for generic developers to purchase samples of a brand-name drug.
“In general, generic drug developers need the samples of the brand drug to develop their generic product and/or to conduct testing to show that their product is bioequivalent to the brand drug for FDA approval,” he said.
“Without these samples, generic drug makers may not be able to develop generic alternatives. Yet the FDA has heard that some brand companies will adopt tactics to make it hard for the generic companies to purchase these brand drugs at a fair value and in the open marketplace.”
One such tactic is for brand companies to place restrictions in their commercial contracts or agreements with prescription drug distributors, wholesalers, or specialty pharmacies that limit their ability to sell samples to generic drug developers.
In other cases, brand companies may be preventing generic drug developers from accessing samples due to limited distribution programs required by a Risk Evaluation and Mitigation Strategy (REMS) with Elements to Assure Safe Use (ETASU).
“But I want to be very clear: a path to securing samples of brand drugs for the purpose of generic drug development should always be available,” Dr Gottlieb said. “Even in the case of limited distribution programs, such as those required by certain REMS, there should be a path forward for generic drug development.”
With this in mind, the FDA has released a list of brand companies that may have prevented generic drug developers from accessing samples. The list contains 52 brand-name drugs, including Pomalyst, Promacta, Revlimid, Tasigna, and Thalidomid.
Generic drug developers have reported trouble obtaining samples of these drugs and asked the FDA for assistance.
When the FDA receives such a request, the agency first determines whether the product in question has a REMS program with ETASU that may impact distribution.
If the FDA confirms the existence of such a program, the generic company can submit its bioequivalence testing protocol to the FDA. The agency then evaluates this protocol to ensure the plan for testing the product contains safety protections comparable to the brand product’s REMS program.
If a generic developer’s plans include appropriate protections, the developer can request that the FDA send a Safety Determination Letter to the brand company. This letter notes that the REMS program doesn’t prevent the brand company from selling its product to generic drug developers.
The FDA has issued 21 Safety Determination Letters to date, according to Dr Gottlieb.
If a generic drug developer has trouble obtaining samples of a drug for which there is no REMS program with ETASU, the FDA informs the developer that there are no FDA-required restrictions that would prevent it from obtaining samples.
The FDA also notifies the Federal Trade Commission (FTC) of requests from generic drug developers seeking assistance in obtaining samples from brand companies. And the FDA encourages generic drug developers to raise these cases with the FTC if they believe that anticompetitive conduct has taken place.
The US Food and Drug Administration (FDA) has released a list of companies that “may be pursuing gaming tactics to delay generic competition.”
FDA Commissioner Scott Gottlieb, MD, said the agency is taking steps to improve access to drugs and make them more affordable.
One of these steps is trying to prevent companies that market brand-name drugs from delaying the release of generic equivalents.
Dr Gottlieb explained that brand companies may create obstacles for generic developers to purchase samples of a brand-name drug.
“In general, generic drug developers need the samples of the brand drug to develop their generic product and/or to conduct testing to show that their product is bioequivalent to the brand drug for FDA approval,” he said.
“Without these samples, generic drug makers may not be able to develop generic alternatives. Yet the FDA has heard that some brand companies will adopt tactics to make it hard for the generic companies to purchase these brand drugs at a fair value and in the open marketplace.”
One such tactic is for brand companies to place restrictions in their commercial contracts or agreements with prescription drug distributors, wholesalers, or specialty pharmacies that limit their ability to sell samples to generic drug developers.
In other cases, brand companies may be preventing generic drug developers from accessing samples due to limited distribution programs required by a Risk Evaluation and Mitigation Strategy (REMS) with Elements to Assure Safe Use (ETASU).
“But I want to be very clear: a path to securing samples of brand drugs for the purpose of generic drug development should always be available,” Dr Gottlieb said. “Even in the case of limited distribution programs, such as those required by certain REMS, there should be a path forward for generic drug development.”
With this in mind, the FDA has released a list of brand companies that may have prevented generic drug developers from accessing samples. The list contains 52 brand-name drugs, including Pomalyst, Promacta, Revlimid, Tasigna, and Thalidomid.
Generic drug developers have reported trouble obtaining samples of these drugs and asked the FDA for assistance.
When the FDA receives such a request, the agency first determines whether the product in question has a REMS program with ETASU that may impact distribution.
If the FDA confirms the existence of such a program, the generic company can submit its bioequivalence testing protocol to the FDA. The agency then evaluates this protocol to ensure the plan for testing the product contains safety protections comparable to the brand product’s REMS program.
If a generic developer’s plans include appropriate protections, the developer can request that the FDA send a Safety Determination Letter to the brand company. This letter notes that the REMS program doesn’t prevent the brand company from selling its product to generic drug developers.
The FDA has issued 21 Safety Determination Letters to date, according to Dr Gottlieb.
If a generic drug developer has trouble obtaining samples of a drug for which there is no REMS program with ETASU, the FDA informs the developer that there are no FDA-required restrictions that would prevent it from obtaining samples.
The FDA also notifies the Federal Trade Commission (FTC) of requests from generic drug developers seeking assistance in obtaining samples from brand companies. And the FDA encourages generic drug developers to raise these cases with the FTC if they believe that anticompetitive conduct has taken place.
FDA approves first epoetin alfa biosimilar
The US Food and Drug Administration (FDA) has approved epoetin alfa-epbx (Retacrit), a biosimilar to epoetin alfa (Epogen/Procrit).
Epoetin alfa-epbx is approved for the treatment of anemia caused by chronic kidney disease, the use of zidovudine in patients with HIV infection, and myelosuppressive chemotherapy in patients who have a minimum of 2 additional months of planned chemotherapy.
Epoetin alfa-epbx is also approved for use before and after surgery to reduce the chance that red blood cell transfusions will be needed because of blood loss during elective, noncardiac, or nonvascular surgery.
As with epoetin alfa, the prescribing information for epoetin alfa-epbx contains a Boxed Warning noting that erythropoiesis-stimulating agents increase the risk of death, myocardial infarction, stroke, venous thromboembolism, thrombosis of vascular access, and tumor progression or recurrence.
The FDA granted approval of epoetin alfa-epbx to Hospira Inc., a Pfizer company.
The agency’s approval is based on a review of evidence that included structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamic data, clinical immunogenicity data, and other clinical safety and effectiveness data.
This evidence is available in an FDA briefing document on the biologics license application for epoetin alfa-epbx.
The US Food and Drug Administration (FDA) has approved epoetin alfa-epbx (Retacrit), a biosimilar to epoetin alfa (Epogen/Procrit).
Epoetin alfa-epbx is approved for the treatment of anemia caused by chronic kidney disease, the use of zidovudine in patients with HIV infection, and myelosuppressive chemotherapy in patients who have a minimum of 2 additional months of planned chemotherapy.
Epoetin alfa-epbx is also approved for use before and after surgery to reduce the chance that red blood cell transfusions will be needed because of blood loss during elective, noncardiac, or nonvascular surgery.
As with epoetin alfa, the prescribing information for epoetin alfa-epbx contains a Boxed Warning noting that erythropoiesis-stimulating agents increase the risk of death, myocardial infarction, stroke, venous thromboembolism, thrombosis of vascular access, and tumor progression or recurrence.
The FDA granted approval of epoetin alfa-epbx to Hospira Inc., a Pfizer company.
The agency’s approval is based on a review of evidence that included structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamic data, clinical immunogenicity data, and other clinical safety and effectiveness data.
This evidence is available in an FDA briefing document on the biologics license application for epoetin alfa-epbx.
The US Food and Drug Administration (FDA) has approved epoetin alfa-epbx (Retacrit), a biosimilar to epoetin alfa (Epogen/Procrit).
Epoetin alfa-epbx is approved for the treatment of anemia caused by chronic kidney disease, the use of zidovudine in patients with HIV infection, and myelosuppressive chemotherapy in patients who have a minimum of 2 additional months of planned chemotherapy.
Epoetin alfa-epbx is also approved for use before and after surgery to reduce the chance that red blood cell transfusions will be needed because of blood loss during elective, noncardiac, or nonvascular surgery.
As with epoetin alfa, the prescribing information for epoetin alfa-epbx contains a Boxed Warning noting that erythropoiesis-stimulating agents increase the risk of death, myocardial infarction, stroke, venous thromboembolism, thrombosis of vascular access, and tumor progression or recurrence.
The FDA granted approval of epoetin alfa-epbx to Hospira Inc., a Pfizer company.
The agency’s approval is based on a review of evidence that included structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamic data, clinical immunogenicity data, and other clinical safety and effectiveness data.
This evidence is available in an FDA briefing document on the biologics license application for epoetin alfa-epbx.
Galinpepimut-S receives orphan designation for MM
The US Food and Drug Administration (FDA) has granted orphan drug designation to galinpepimut-S (GPS) as a treatment for multiple myeloma (MM).
GPS is an immunotherapeutic that targets malignancies characterized by overexpression of the Wilms tumor 1 (WT1) antigen.
GPS consists of 4 peptide chains, 2 of which are modified chains that induce an innate immune response (CD4+/CD8+) against the WT1 antigen and access a range of HLA types.
When GPS is administered to a patient, the induced immune response has the potential to recognize and destroy cancer cells and provide ongoing support to the immune system so it can continue to target and destroy residual cancer cells.
GPS also has orphan designation from the FDA for the treatment of acute myeloid leukemia and malignant plural mesothelioma.
Phase 2 trial
GPS has been investigated in a phase 2 trial of MM patients. Results from this trial were recently presented at the 44th Annual Meeting of the EBMT.
Researchers evaluated GPS in combination with lenalidomide as maintenance therapy in MM patients who received an autologous stem cell transplant (ASCT).
The study enrolled 19 patients who began receiving GPS within 22 days of ASCT. They received 6 doses every 2 weeks. (Injection sites were pre-stimulated with granulocyte-macrophage colony-stimulating factor.)
Patients received 6 additional monthly doses of GPS as well as lenalidomide maintenance (10 mg daily) starting on day 100 post-ASCT.
Twelve patients received all 12 doses of GPS. Eleven patients achieved a complete response or very good partial response. All of these patients had CD4 immune responses, and 9 of them had CD8 immune responses.
The progression-free survival was 81% at 12 months and 62% at 18 months. The median progression-free survival was 23.6 months (range, 15.2 to not reached).
The overall survival was 88% at 18 months, and the median overall survival was not reached.
About orphan designation
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 US Food and Drug Administration (FDA) has granted orphan drug designation to galinpepimut-S (GPS) as a treatment for multiple myeloma (MM).
GPS is an immunotherapeutic that targets malignancies characterized by overexpression of the Wilms tumor 1 (WT1) antigen.
GPS consists of 4 peptide chains, 2 of which are modified chains that induce an innate immune response (CD4+/CD8+) against the WT1 antigen and access a range of HLA types.
When GPS is administered to a patient, the induced immune response has the potential to recognize and destroy cancer cells and provide ongoing support to the immune system so it can continue to target and destroy residual cancer cells.
GPS also has orphan designation from the FDA for the treatment of acute myeloid leukemia and malignant plural mesothelioma.
Phase 2 trial
GPS has been investigated in a phase 2 trial of MM patients. Results from this trial were recently presented at the 44th Annual Meeting of the EBMT.
Researchers evaluated GPS in combination with lenalidomide as maintenance therapy in MM patients who received an autologous stem cell transplant (ASCT).
The study enrolled 19 patients who began receiving GPS within 22 days of ASCT. They received 6 doses every 2 weeks. (Injection sites were pre-stimulated with granulocyte-macrophage colony-stimulating factor.)
Patients received 6 additional monthly doses of GPS as well as lenalidomide maintenance (10 mg daily) starting on day 100 post-ASCT.
Twelve patients received all 12 doses of GPS. Eleven patients achieved a complete response or very good partial response. All of these patients had CD4 immune responses, and 9 of them had CD8 immune responses.
The progression-free survival was 81% at 12 months and 62% at 18 months. The median progression-free survival was 23.6 months (range, 15.2 to not reached).
The overall survival was 88% at 18 months, and the median overall survival was not reached.
About orphan designation
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 US Food and Drug Administration (FDA) has granted orphan drug designation to galinpepimut-S (GPS) as a treatment for multiple myeloma (MM).
GPS is an immunotherapeutic that targets malignancies characterized by overexpression of the Wilms tumor 1 (WT1) antigen.
GPS consists of 4 peptide chains, 2 of which are modified chains that induce an innate immune response (CD4+/CD8+) against the WT1 antigen and access a range of HLA types.
When GPS is administered to a patient, the induced immune response has the potential to recognize and destroy cancer cells and provide ongoing support to the immune system so it can continue to target and destroy residual cancer cells.
GPS also has orphan designation from the FDA for the treatment of acute myeloid leukemia and malignant plural mesothelioma.
Phase 2 trial
GPS has been investigated in a phase 2 trial of MM patients. Results from this trial were recently presented at the 44th Annual Meeting of the EBMT.
Researchers evaluated GPS in combination with lenalidomide as maintenance therapy in MM patients who received an autologous stem cell transplant (ASCT).
The study enrolled 19 patients who began receiving GPS within 22 days of ASCT. They received 6 doses every 2 weeks. (Injection sites were pre-stimulated with granulocyte-macrophage colony-stimulating factor.)
Patients received 6 additional monthly doses of GPS as well as lenalidomide maintenance (10 mg daily) starting on day 100 post-ASCT.
Twelve patients received all 12 doses of GPS. Eleven patients achieved a complete response or very good partial response. All of these patients had CD4 immune responses, and 9 of them had CD8 immune responses.
The progression-free survival was 81% at 12 months and 62% at 18 months. The median progression-free survival was 23.6 months (range, 15.2 to not reached).
The overall survival was 88% at 18 months, and the median overall survival was not reached.
About orphan designation
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.