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FDA alert: pembrolizumab ups mortality risk in multiple myeloma
The use of pembrolizumab (Keytruda) in combination with dexamethasone and either lenalidomide or pomalidomide is associated with an increased risk of death in patients with multiple myeloma, according to an alert from the Food and Drug Administration.
The FDA issued the alert on Aug. 31 to inform the public, health care professionals, and oncology clinical investigators of the risk, which became apparent during the course of two now-halted clinical trials.
Among 249 patients in KEYNOTE-183, which evaluated pomalidomide and low-dose dexamethasone with or without pembrolizumab in patients with relapsed and refractory multiple myeloma who had received at least two prior lines of therapy, the relative risk of death was increased more than 50% among those in the pembrolizumab-containing investigational arm, compared with the control arm (hazard ratio, 1.61).
Among 301 patients in KEYNOTE-185, which evaluated lenalidomide and low-dose dexamethasone with or without pembrolizumab in patients with newly diagnosed multiple myeloma who were ineligible for autologous stem cell transplant, the relative risk of death was nearly doubled in the pembrolizumab-containing investigational arm, compared with the control arm (hazard ratio, 2.06).
“On July 3, 2017, the FDA required that all patients in these trials be discontinued from further investigation with this drug, because interim results from both trials demonstrated an increased risk of death for patients receiving Keytruda when it was combined with an immunomodulatory agent as compared to the control group,” according to the statement. “Merck & Co., Inc. [the maker of Keytruda] was made aware of the issue through an external data monitoring committee recommendation and suspended the trials to enrollment on June 12, 2017.”
The FDA statement and actions by Merck do not apply to patients using the drug for approved indications, which include melanoma, lung cancer, head and neck cancer, classical Hodgkin lymphoma, urothelial carcinoma, and microsatellite instability–high (MSI-H) cancer.
“Patients on Keytruda for an approved use should continue to take their medication as directed by their health care professional,” according to the FDA, which noted in its statement that the agency will be working directly with sponsors and investigators of other ongoing trials of the drug and other PD-1/PD-L1 cancer drugs to “determine the extent of safety issues.”
In a separate statement, Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research further stressed that the FDA is “actively examining the data from the Keytruda trials and working directly with Merck to better understand the true cause of the safety concerns.”
“The FDA will take appropriate action as warranted to ensure patients enrolled in these trials are protected and that doctors and clinical trial researchers understand the risks associated with this investigational use. We are communicating now, given the serious nature of the safety issue, to remind doctors and patients that Keytruda is not approved for the treatment of multiple myeloma and should not be given to patients in combination with any immunomodulatory agents, including Revlimid (lenalidomide) and Pomalyst (pomalidomide), for the treatment of multiple myeloma,” she said.
Health care professionals and consumers are encouraged to report adverse events and side effects related to Keytruda or similar product to FDA’s MedWatch Adverse Event Reporting program at www.fda.gov/MedWatch/report, or by calling 800-332-1088 to request a reporting form.
sworcester@frontlinemedcom.com
The use of pembrolizumab (Keytruda) in combination with dexamethasone and either lenalidomide or pomalidomide is associated with an increased risk of death in patients with multiple myeloma, according to an alert from the Food and Drug Administration.
The FDA issued the alert on Aug. 31 to inform the public, health care professionals, and oncology clinical investigators of the risk, which became apparent during the course of two now-halted clinical trials.
Among 249 patients in KEYNOTE-183, which evaluated pomalidomide and low-dose dexamethasone with or without pembrolizumab in patients with relapsed and refractory multiple myeloma who had received at least two prior lines of therapy, the relative risk of death was increased more than 50% among those in the pembrolizumab-containing investigational arm, compared with the control arm (hazard ratio, 1.61).
Among 301 patients in KEYNOTE-185, which evaluated lenalidomide and low-dose dexamethasone with or without pembrolizumab in patients with newly diagnosed multiple myeloma who were ineligible for autologous stem cell transplant, the relative risk of death was nearly doubled in the pembrolizumab-containing investigational arm, compared with the control arm (hazard ratio, 2.06).
“On July 3, 2017, the FDA required that all patients in these trials be discontinued from further investigation with this drug, because interim results from both trials demonstrated an increased risk of death for patients receiving Keytruda when it was combined with an immunomodulatory agent as compared to the control group,” according to the statement. “Merck & Co., Inc. [the maker of Keytruda] was made aware of the issue through an external data monitoring committee recommendation and suspended the trials to enrollment on June 12, 2017.”
The FDA statement and actions by Merck do not apply to patients using the drug for approved indications, which include melanoma, lung cancer, head and neck cancer, classical Hodgkin lymphoma, urothelial carcinoma, and microsatellite instability–high (MSI-H) cancer.
“Patients on Keytruda for an approved use should continue to take their medication as directed by their health care professional,” according to the FDA, which noted in its statement that the agency will be working directly with sponsors and investigators of other ongoing trials of the drug and other PD-1/PD-L1 cancer drugs to “determine the extent of safety issues.”
In a separate statement, Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research further stressed that the FDA is “actively examining the data from the Keytruda trials and working directly with Merck to better understand the true cause of the safety concerns.”
“The FDA will take appropriate action as warranted to ensure patients enrolled in these trials are protected and that doctors and clinical trial researchers understand the risks associated with this investigational use. We are communicating now, given the serious nature of the safety issue, to remind doctors and patients that Keytruda is not approved for the treatment of multiple myeloma and should not be given to patients in combination with any immunomodulatory agents, including Revlimid (lenalidomide) and Pomalyst (pomalidomide), for the treatment of multiple myeloma,” she said.
Health care professionals and consumers are encouraged to report adverse events and side effects related to Keytruda or similar product to FDA’s MedWatch Adverse Event Reporting program at www.fda.gov/MedWatch/report, or by calling 800-332-1088 to request a reporting form.
sworcester@frontlinemedcom.com
The use of pembrolizumab (Keytruda) in combination with dexamethasone and either lenalidomide or pomalidomide is associated with an increased risk of death in patients with multiple myeloma, according to an alert from the Food and Drug Administration.
The FDA issued the alert on Aug. 31 to inform the public, health care professionals, and oncology clinical investigators of the risk, which became apparent during the course of two now-halted clinical trials.
Among 249 patients in KEYNOTE-183, which evaluated pomalidomide and low-dose dexamethasone with or without pembrolizumab in patients with relapsed and refractory multiple myeloma who had received at least two prior lines of therapy, the relative risk of death was increased more than 50% among those in the pembrolizumab-containing investigational arm, compared with the control arm (hazard ratio, 1.61).
Among 301 patients in KEYNOTE-185, which evaluated lenalidomide and low-dose dexamethasone with or without pembrolizumab in patients with newly diagnosed multiple myeloma who were ineligible for autologous stem cell transplant, the relative risk of death was nearly doubled in the pembrolizumab-containing investigational arm, compared with the control arm (hazard ratio, 2.06).
“On July 3, 2017, the FDA required that all patients in these trials be discontinued from further investigation with this drug, because interim results from both trials demonstrated an increased risk of death for patients receiving Keytruda when it was combined with an immunomodulatory agent as compared to the control group,” according to the statement. “Merck & Co., Inc. [the maker of Keytruda] was made aware of the issue through an external data monitoring committee recommendation and suspended the trials to enrollment on June 12, 2017.”
The FDA statement and actions by Merck do not apply to patients using the drug for approved indications, which include melanoma, lung cancer, head and neck cancer, classical Hodgkin lymphoma, urothelial carcinoma, and microsatellite instability–high (MSI-H) cancer.
“Patients on Keytruda for an approved use should continue to take their medication as directed by their health care professional,” according to the FDA, which noted in its statement that the agency will be working directly with sponsors and investigators of other ongoing trials of the drug and other PD-1/PD-L1 cancer drugs to “determine the extent of safety issues.”
In a separate statement, Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research further stressed that the FDA is “actively examining the data from the Keytruda trials and working directly with Merck to better understand the true cause of the safety concerns.”
“The FDA will take appropriate action as warranted to ensure patients enrolled in these trials are protected and that doctors and clinical trial researchers understand the risks associated with this investigational use. We are communicating now, given the serious nature of the safety issue, to remind doctors and patients that Keytruda is not approved for the treatment of multiple myeloma and should not be given to patients in combination with any immunomodulatory agents, including Revlimid (lenalidomide) and Pomalyst (pomalidomide), for the treatment of multiple myeloma,” she said.
Health care professionals and consumers are encouraged to report adverse events and side effects related to Keytruda or similar product to FDA’s MedWatch Adverse Event Reporting program at www.fda.gov/MedWatch/report, or by calling 800-332-1088 to request a reporting form.
sworcester@frontlinemedcom.com
Austedo approved for treatment of tardive dyskinesia
The Food and Drug Administration has approved deutetrabenazine (Austedo) for the treatment of tardive dyskinesia in adults, according to an announcement from Teva Pharmaceutical Industries.
The agency’s approval of Austedo was based on results from two phase 3 clinical trials in which the drug was shown to be safe and effective at reducing involuntary movements collectively termed tardive dyskinesia, a debilitating and sometimes irreversible movement disorder which affects about 500,000 people in the United States. Austedo was first approved in April 2017 to treat chorea associated with Huntington’s disease.
“Physicians treating tardive dyskinesia will appreciate the therapy’s dosing flexibility and the ability to focus on directly treating the movement disorder and not disrupt the ongoing treatment for the underlying condition,” Michael Hayden, MD, PhD, President of Global R&D and Chief Scientific Officer at Teva, said in the announcement.
The full prescribing information can be viewed here.
The Food and Drug Administration has approved deutetrabenazine (Austedo) for the treatment of tardive dyskinesia in adults, according to an announcement from Teva Pharmaceutical Industries.
The agency’s approval of Austedo was based on results from two phase 3 clinical trials in which the drug was shown to be safe and effective at reducing involuntary movements collectively termed tardive dyskinesia, a debilitating and sometimes irreversible movement disorder which affects about 500,000 people in the United States. Austedo was first approved in April 2017 to treat chorea associated with Huntington’s disease.
“Physicians treating tardive dyskinesia will appreciate the therapy’s dosing flexibility and the ability to focus on directly treating the movement disorder and not disrupt the ongoing treatment for the underlying condition,” Michael Hayden, MD, PhD, President of Global R&D and Chief Scientific Officer at Teva, said in the announcement.
The full prescribing information can be viewed here.
The Food and Drug Administration has approved deutetrabenazine (Austedo) for the treatment of tardive dyskinesia in adults, according to an announcement from Teva Pharmaceutical Industries.
The agency’s approval of Austedo was based on results from two phase 3 clinical trials in which the drug was shown to be safe and effective at reducing involuntary movements collectively termed tardive dyskinesia, a debilitating and sometimes irreversible movement disorder which affects about 500,000 people in the United States. Austedo was first approved in April 2017 to treat chorea associated with Huntington’s disease.
“Physicians treating tardive dyskinesia will appreciate the therapy’s dosing flexibility and the ability to focus on directly treating the movement disorder and not disrupt the ongoing treatment for the underlying condition,” Michael Hayden, MD, PhD, President of Global R&D and Chief Scientific Officer at Teva, said in the announcement.
The full prescribing information can be viewed here.
FDA approves Vabomere for complicated UTI in adults
The Food and Drug Administration has approved Vabomere (meropenem and vaborbactam) for adults with complicated urinary tract infection (cUTI), including pyelonephritis caused by susceptible Enterobacteriaceae, the agency has announced.
The approval was based on results of the TANGO 1 trial, a phase 3, multicenter, double-blind study of 545 adult patients with cUTI. Overall, 98.4% of patients treated with Vabomere saw improvement in symptoms and negative urine culture tests by the end of intravenous treatment, compared with 94.3% of those treated with piperacillin/tazobactam (95% confidence interval, 0.3%-8.8%). Improvement continued in about 77% of patients treated with Vabomere who had resolved symptoms 7 days after completing treatment, compared with about 73% of those who were treated with piperacillin/tazobactam, the FDA said Aug. 29 in a press release.
Headache, infusion site reactions, and diarrhea were common adverse effects of Vabomere. The drug also has been associated with allergic reactions and seizures, so it should not be administered to patients with a history of anaphylaxis.
“Vabomere represents a significant new advancement in addressing [Klebsiella pneumoniae carbapenemase]-producing Enterobacteriaceae, for which there are currently limited treatment options,” Clive A. Meanwell, MD, PhD, chief executive officer of The Medicines Company, said in the statement.
Rempex Pharmaceuticals, a Medicines Company unit, received the approval. The Medicines Company said the drug is expected to be available before the end of the year.
The Food and Drug Administration has approved Vabomere (meropenem and vaborbactam) for adults with complicated urinary tract infection (cUTI), including pyelonephritis caused by susceptible Enterobacteriaceae, the agency has announced.
The approval was based on results of the TANGO 1 trial, a phase 3, multicenter, double-blind study of 545 adult patients with cUTI. Overall, 98.4% of patients treated with Vabomere saw improvement in symptoms and negative urine culture tests by the end of intravenous treatment, compared with 94.3% of those treated with piperacillin/tazobactam (95% confidence interval, 0.3%-8.8%). Improvement continued in about 77% of patients treated with Vabomere who had resolved symptoms 7 days after completing treatment, compared with about 73% of those who were treated with piperacillin/tazobactam, the FDA said Aug. 29 in a press release.
Headache, infusion site reactions, and diarrhea were common adverse effects of Vabomere. The drug also has been associated with allergic reactions and seizures, so it should not be administered to patients with a history of anaphylaxis.
“Vabomere represents a significant new advancement in addressing [Klebsiella pneumoniae carbapenemase]-producing Enterobacteriaceae, for which there are currently limited treatment options,” Clive A. Meanwell, MD, PhD, chief executive officer of The Medicines Company, said in the statement.
Rempex Pharmaceuticals, a Medicines Company unit, received the approval. The Medicines Company said the drug is expected to be available before the end of the year.
The Food and Drug Administration has approved Vabomere (meropenem and vaborbactam) for adults with complicated urinary tract infection (cUTI), including pyelonephritis caused by susceptible Enterobacteriaceae, the agency has announced.
The approval was based on results of the TANGO 1 trial, a phase 3, multicenter, double-blind study of 545 adult patients with cUTI. Overall, 98.4% of patients treated with Vabomere saw improvement in symptoms and negative urine culture tests by the end of intravenous treatment, compared with 94.3% of those treated with piperacillin/tazobactam (95% confidence interval, 0.3%-8.8%). Improvement continued in about 77% of patients treated with Vabomere who had resolved symptoms 7 days after completing treatment, compared with about 73% of those who were treated with piperacillin/tazobactam, the FDA said Aug. 29 in a press release.
Headache, infusion site reactions, and diarrhea were common adverse effects of Vabomere. The drug also has been associated with allergic reactions and seizures, so it should not be administered to patients with a history of anaphylaxis.
“Vabomere represents a significant new advancement in addressing [Klebsiella pneumoniae carbapenemase]-producing Enterobacteriaceae, for which there are currently limited treatment options,” Clive A. Meanwell, MD, PhD, chief executive officer of The Medicines Company, said in the statement.
Rempex Pharmaceuticals, a Medicines Company unit, received the approval. The Medicines Company said the drug is expected to be available before the end of the year.
FDA grants breakthrough status to epidermolysis bullosa gene therapy
The U.S. Food and Drug Administration has granted breakthrough therapy status to Abeona Therapeutics’s EB-101 gene therapy program for patients with recessive dystrophic epidermolysis bullosa, according to a company statement.
The breakthrough therapy designation will expedite the phase 3 trial, intended to start in 2018, and the approval process and make this therapy available to patients who have few or no other treatment options for this serious and painful condition.
The experimental treatment “utilizes a patient’s own cells and genetically engineer[s] them to produce the correct version of collagen, which helps hold skin on to the body, thereby reducing the number of painful blisters caused by injury and improving wound healing,” Timothy J. Miller, PhD, CEO and president of Abeona, said in a statement. “We are grateful that the FDA has recognized the promising clinical data from the EB-101 program with Breakthrough Therapy designation.”
The U.S. Food and Drug Administration has granted breakthrough therapy status to Abeona Therapeutics’s EB-101 gene therapy program for patients with recessive dystrophic epidermolysis bullosa, according to a company statement.
The breakthrough therapy designation will expedite the phase 3 trial, intended to start in 2018, and the approval process and make this therapy available to patients who have few or no other treatment options for this serious and painful condition.
The experimental treatment “utilizes a patient’s own cells and genetically engineer[s] them to produce the correct version of collagen, which helps hold skin on to the body, thereby reducing the number of painful blisters caused by injury and improving wound healing,” Timothy J. Miller, PhD, CEO and president of Abeona, said in a statement. “We are grateful that the FDA has recognized the promising clinical data from the EB-101 program with Breakthrough Therapy designation.”
The U.S. Food and Drug Administration has granted breakthrough therapy status to Abeona Therapeutics’s EB-101 gene therapy program for patients with recessive dystrophic epidermolysis bullosa, according to a company statement.
The breakthrough therapy designation will expedite the phase 3 trial, intended to start in 2018, and the approval process and make this therapy available to patients who have few or no other treatment options for this serious and painful condition.
The experimental treatment “utilizes a patient’s own cells and genetically engineer[s] them to produce the correct version of collagen, which helps hold skin on to the body, thereby reducing the number of painful blisters caused by injury and improving wound healing,” Timothy J. Miller, PhD, CEO and president of Abeona, said in a statement. “We are grateful that the FDA has recognized the promising clinical data from the EB-101 program with Breakthrough Therapy designation.”
FDA grants accelerated approval for treatment of Chagas disease in children
The Food and Drug Administration announced Aug. 29 the accelerated approval of benznidazole for use in children aged 2-12 years who have Chagas disease.
In two placebo-controlled clinical trials, researchers examined pediatric patients aged 6-12 with Chagas disease. In the first trial, about 60% of children treated with benznidazole had an antibody test change from positive to negative, compared with about 14% children who received a placebo. Similar findings were found in the second trial. In that one, about 55% of children treated with benznidazole had an antibody test change from positive to negative, compared with 5% who received a placebo. Also, a different study of the safety and pharmacokinetics of benznidazole in pediatric patients aged 2-12 provided information for dosing recommendations down to 2 years of age.
“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
This is the first treatment approved in the United States for Chagas disease. According to the agency, additional study is needed to “verify and describe the anticipated clinical benefit of benznidazole.”
Read the full press release on the FDA’s website.
The Food and Drug Administration announced Aug. 29 the accelerated approval of benznidazole for use in children aged 2-12 years who have Chagas disease.
In two placebo-controlled clinical trials, researchers examined pediatric patients aged 6-12 with Chagas disease. In the first trial, about 60% of children treated with benznidazole had an antibody test change from positive to negative, compared with about 14% children who received a placebo. Similar findings were found in the second trial. In that one, about 55% of children treated with benznidazole had an antibody test change from positive to negative, compared with 5% who received a placebo. Also, a different study of the safety and pharmacokinetics of benznidazole in pediatric patients aged 2-12 provided information for dosing recommendations down to 2 years of age.
“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
This is the first treatment approved in the United States for Chagas disease. According to the agency, additional study is needed to “verify and describe the anticipated clinical benefit of benznidazole.”
Read the full press release on the FDA’s website.
The Food and Drug Administration announced Aug. 29 the accelerated approval of benznidazole for use in children aged 2-12 years who have Chagas disease.
In two placebo-controlled clinical trials, researchers examined pediatric patients aged 6-12 with Chagas disease. In the first trial, about 60% of children treated with benznidazole had an antibody test change from positive to negative, compared with about 14% children who received a placebo. Similar findings were found in the second trial. In that one, about 55% of children treated with benznidazole had an antibody test change from positive to negative, compared with 5% who received a placebo. Also, a different study of the safety and pharmacokinetics of benznidazole in pediatric patients aged 2-12 provided information for dosing recommendations down to 2 years of age.
“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
This is the first treatment approved in the United States for Chagas disease. According to the agency, additional study is needed to “verify and describe the anticipated clinical benefit of benznidazole.”
Read the full press release on the FDA’s website.
FDA expands approval of fulvestrant for HR+/HER2– advanced breast cancer
The Food and Drug Administration has approved fulvestrant (Faslodex) as monotherapy for women with hormone receptor–positive, human epidermal growth factor receptor 2–negative (HR+/HER2–) advanced breast cancer who are postmenopausal and previously untreated.
Approval was based on improved progression-free survival (PFS) in the phase 3 FALCON trial of 462 postmenopausal women with HR+/HER2– metastatic or locally advanced breast cancer who had not previously received hormonal therapy, drug maker AstraZeneca said in a press release.
The most common adverse events were arthralgia (16.7% of patients on fulvestrant vs. 10.3% on anastrozole), and hot flushes/flashes (11.4% vs. 10.3%).
Grade 3 or greater adverse events occurred in 22.4% vs. 17.7%. Deaths from adverse events occurred in six patients on fulvestrant vs. seven on anastrozole, according to results published in The Lancet.
The Food and Drug Administration has approved fulvestrant (Faslodex) as monotherapy for women with hormone receptor–positive, human epidermal growth factor receptor 2–negative (HR+/HER2–) advanced breast cancer who are postmenopausal and previously untreated.
Approval was based on improved progression-free survival (PFS) in the phase 3 FALCON trial of 462 postmenopausal women with HR+/HER2– metastatic or locally advanced breast cancer who had not previously received hormonal therapy, drug maker AstraZeneca said in a press release.
The most common adverse events were arthralgia (16.7% of patients on fulvestrant vs. 10.3% on anastrozole), and hot flushes/flashes (11.4% vs. 10.3%).
Grade 3 or greater adverse events occurred in 22.4% vs. 17.7%. Deaths from adverse events occurred in six patients on fulvestrant vs. seven on anastrozole, according to results published in The Lancet.
The Food and Drug Administration has approved fulvestrant (Faslodex) as monotherapy for women with hormone receptor–positive, human epidermal growth factor receptor 2–negative (HR+/HER2–) advanced breast cancer who are postmenopausal and previously untreated.
Approval was based on improved progression-free survival (PFS) in the phase 3 FALCON trial of 462 postmenopausal women with HR+/HER2– metastatic or locally advanced breast cancer who had not previously received hormonal therapy, drug maker AstraZeneca said in a press release.
The most common adverse events were arthralgia (16.7% of patients on fulvestrant vs. 10.3% on anastrozole), and hot flushes/flashes (11.4% vs. 10.3%).
Grade 3 or greater adverse events occurred in 22.4% vs. 17.7%. Deaths from adverse events occurred in six patients on fulvestrant vs. seven on anastrozole, according to results published in The Lancet.
FDA approves first gene therapy – tisagenlecleucel for ALL
The U.S. Food and Drug Administration has approved tisagenlecleucel (Kymriah), a first-of-its-kind chimeric antigen receptor T-cell (CAR T) therapy, for the treatment of children and young adults up to age 25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
Tisagenlecleucel will carry a boxed warning regarding the CRS risk. Additionally, due to the CRS risk and risk of neurological events, the approval requires a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use, according to an FDA press release.
Special certification will be required for hospitals and associated clinics that dispense tisagenlecleucel. As part of certification, staff will be trained in the prescribing, dispensing, or administering of the therapy, and to recognize and manage CRS and neurological events.
Novartis, the maker of tisagenlecleucel, will be required to conduct postmarketing observational study.
Indeed, FDA commissioner Scott Gottlieb, MD, said the approval marks the entry to a “new frontier in medical innovation.”
“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses,” he said in the press statement.
Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy involving customized treatment created using a patient’s own T cells. The T cells are genetically modified to include a chimeric antigen receptor that directs the T cells to target and kill leukemia cells with CD19 surface antigen, and are then infused back into the patient.
In a phase 2 clinical trial, the overall remission rate with tisagenlecleucel therapy was 83% in 63 children and young adults with relapsed/refractory B-cell precursor ALL for whom at least two prior lines of therapy had failed; the therapy was granted Fast Track, Priority Review, and Breakthrough Therapy designations.
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research said in the press statement.
“Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that shows promising remission and survival rates in clinical trials.”
At its meeting in July, the FDA ODAC agreed nearly unanimously that the risk mitigation plan put forward by Novartis, including planned 15-year follow-up and other mitigation measures, would be adequate for detecting serious consequences of CAR T-cell therapy.
sworcester@frontlinemedcom.com
This article was updated August 30, 2017.
The U.S. Food and Drug Administration has approved tisagenlecleucel (Kymriah), a first-of-its-kind chimeric antigen receptor T-cell (CAR T) therapy, for the treatment of children and young adults up to age 25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
Tisagenlecleucel will carry a boxed warning regarding the CRS risk. Additionally, due to the CRS risk and risk of neurological events, the approval requires a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use, according to an FDA press release.
Special certification will be required for hospitals and associated clinics that dispense tisagenlecleucel. As part of certification, staff will be trained in the prescribing, dispensing, or administering of the therapy, and to recognize and manage CRS and neurological events.
Novartis, the maker of tisagenlecleucel, will be required to conduct postmarketing observational study.
Indeed, FDA commissioner Scott Gottlieb, MD, said the approval marks the entry to a “new frontier in medical innovation.”
“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses,” he said in the press statement.
Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy involving customized treatment created using a patient’s own T cells. The T cells are genetically modified to include a chimeric antigen receptor that directs the T cells to target and kill leukemia cells with CD19 surface antigen, and are then infused back into the patient.
In a phase 2 clinical trial, the overall remission rate with tisagenlecleucel therapy was 83% in 63 children and young adults with relapsed/refractory B-cell precursor ALL for whom at least two prior lines of therapy had failed; the therapy was granted Fast Track, Priority Review, and Breakthrough Therapy designations.
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research said in the press statement.
“Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that shows promising remission and survival rates in clinical trials.”
At its meeting in July, the FDA ODAC agreed nearly unanimously that the risk mitigation plan put forward by Novartis, including planned 15-year follow-up and other mitigation measures, would be adequate for detecting serious consequences of CAR T-cell therapy.
sworcester@frontlinemedcom.com
This article was updated August 30, 2017.
The U.S. Food and Drug Administration has approved tisagenlecleucel (Kymriah), a first-of-its-kind chimeric antigen receptor T-cell (CAR T) therapy, for the treatment of children and young adults up to age 25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
Tisagenlecleucel will carry a boxed warning regarding the CRS risk. Additionally, due to the CRS risk and risk of neurological events, the approval requires a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use, according to an FDA press release.
Special certification will be required for hospitals and associated clinics that dispense tisagenlecleucel. As part of certification, staff will be trained in the prescribing, dispensing, or administering of the therapy, and to recognize and manage CRS and neurological events.
Novartis, the maker of tisagenlecleucel, will be required to conduct postmarketing observational study.
Indeed, FDA commissioner Scott Gottlieb, MD, said the approval marks the entry to a “new frontier in medical innovation.”
“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses,” he said in the press statement.
Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy involving customized treatment created using a patient’s own T cells. The T cells are genetically modified to include a chimeric antigen receptor that directs the T cells to target and kill leukemia cells with CD19 surface antigen, and are then infused back into the patient.
In a phase 2 clinical trial, the overall remission rate with tisagenlecleucel therapy was 83% in 63 children and young adults with relapsed/refractory B-cell precursor ALL for whom at least two prior lines of therapy had failed; the therapy was granted Fast Track, Priority Review, and Breakthrough Therapy designations.
“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research said in the press statement.
“Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that shows promising remission and survival rates in clinical trials.”
At its meeting in July, the FDA ODAC agreed nearly unanimously that the risk mitigation plan put forward by Novartis, including planned 15-year follow-up and other mitigation measures, would be adequate for detecting serious consequences of CAR T-cell therapy.
sworcester@frontlinemedcom.com
This article was updated August 30, 2017.
CDC: Flu vaccine recommendations broaden for pregnant women and children
, according to new recommendations from the Centers for Disease Control and Prevention.
This change from the CDC’s previous guidance that pregnant women receive a seasonal inactivated influenza vaccine (IIV) was recommended by the Advisory Committee on Immunization Practices after some heated debate among committee members over evidence presented to support the change in wording (MMWR. 2017 Aug 25;66[(RR-2]:1-20). 
The new update gives women the ability to choose between receiving an IIV and FluBlok, a recombinant influenza vaccine (RIV) that is not egg based and can be manufactured more quickly, making it ideal in cases of pandemic or supply shortages, according to the CDC.
Although pregnant women may choose to receive a vaccination during the first trimester, the CDC warns there may be some risk involved.
“Although experience with the use of IIVs is substantial, and data from observational studies are available to support the safety of these vaccines in pregnancy, data are more limited for vaccination during the first trimester,” according to the CDC. “Moreover, there is substantially less experience with more recently licensed IIV products (e.g., quadrivalent, cell culture-based, and adjuvanted vaccines) during pregnancy in general.”
Data also are limited regarding RIVs, the CDC said, with the data used to determine safety among pregnant women “limited to reports of pregnancies occurring incidentally during clinical trials, Vaccine Adverse Event Reporting System (VAERS) reports, and pregnancy registry reports.”
Changes for children
The CDC chose to accept ACIP recommendations regarding Afluria (IIV3), expanding the age of children who can receive the vaccine from 9 years and older to 5 years and older.
Similar labeling changes were accepted for FluLaval Quadrivalent (IIV4), which had previously been given to children 3 years and older but now but will be available for children starting at 6 months of age.
New products
Recent product licensures included in the MMWR report are Afluria Quadrivalent (IIV4) and Flublok Quadrivalent (RIV4), both for persons over 18 years.
According to the CDC, Flublok Quadrivalent (an RIV) met noninferiority measures, compared with a similar IIV quadrivalent vaccine, for the A(H3H2) and B/Yamagata viruses but not for A(H1N1) or B/Victoria viruses.
Vaccine composition for 2017-2018
Approved viruses for the 2017-2018 season trivalent vaccines are an A/Michigan/45/2015 (H1N1) pdm09–like virus, an A/Hong Kong/4801/2014 (H3N2)-like virus, and a B/Brisbane/60/2008–like virus (Victoria lineage), according to the MMWR. Quadrivalent vaccines will include those viruses, with the addition of an B/Phuket/3073/2013–like virus (Yamagata lineage).
The CDC continues to recommend that the quadrivalent live attenuated influenza vaccine FluMist not be used by anyone for the 2017-2018 season, a decision that was made after evidence showed poor effectiveness against influenza A(H1N1)pdm09 viruses in the 2013-2014 and 2015-2016 seasons.
Vaccine updates published in this report were recommended by ACIP during meetings held in October 2016 and February and June 2017.
ezimmerman@frontlinemedcom.com
On Twitter @eaztweets
, according to new recommendations from the Centers for Disease Control and Prevention.
This change from the CDC’s previous guidance that pregnant women receive a seasonal inactivated influenza vaccine (IIV) was recommended by the Advisory Committee on Immunization Practices after some heated debate among committee members over evidence presented to support the change in wording (MMWR. 2017 Aug 25;66[(RR-2]:1-20).
The new update gives women the ability to choose between receiving an IIV and FluBlok, a recombinant influenza vaccine (RIV) that is not egg based and can be manufactured more quickly, making it ideal in cases of pandemic or supply shortages, according to the CDC.
Although pregnant women may choose to receive a vaccination during the first trimester, the CDC warns there may be some risk involved.
“Although experience with the use of IIVs is substantial, and data from observational studies are available to support the safety of these vaccines in pregnancy, data are more limited for vaccination during the first trimester,” according to the CDC. “Moreover, there is substantially less experience with more recently licensed IIV products (e.g., quadrivalent, cell culture-based, and adjuvanted vaccines) during pregnancy in general.”
Data also are limited regarding RIVs, the CDC said, with the data used to determine safety among pregnant women “limited to reports of pregnancies occurring incidentally during clinical trials, Vaccine Adverse Event Reporting System (VAERS) reports, and pregnancy registry reports.”
Changes for children
The CDC chose to accept ACIP recommendations regarding Afluria (IIV3), expanding the age of children who can receive the vaccine from 9 years and older to 5 years and older.
Similar labeling changes were accepted for FluLaval Quadrivalent (IIV4), which had previously been given to children 3 years and older but now but will be available for children starting at 6 months of age.
New products
Recent product licensures included in the MMWR report are Afluria Quadrivalent (IIV4) and Flublok Quadrivalent (RIV4), both for persons over 18 years.
According to the CDC, Flublok Quadrivalent (an RIV) met noninferiority measures, compared with a similar IIV quadrivalent vaccine, for the A(H3H2) and B/Yamagata viruses but not for A(H1N1) or B/Victoria viruses.
Vaccine composition for 2017-2018
Approved viruses for the 2017-2018 season trivalent vaccines are an A/Michigan/45/2015 (H1N1) pdm09–like virus, an A/Hong Kong/4801/2014 (H3N2)-like virus, and a B/Brisbane/60/2008–like virus (Victoria lineage), according to the MMWR. Quadrivalent vaccines will include those viruses, with the addition of an B/Phuket/3073/2013–like virus (Yamagata lineage).
The CDC continues to recommend that the quadrivalent live attenuated influenza vaccine FluMist not be used by anyone for the 2017-2018 season, a decision that was made after evidence showed poor effectiveness against influenza A(H1N1)pdm09 viruses in the 2013-2014 and 2015-2016 seasons.
Vaccine updates published in this report were recommended by ACIP during meetings held in October 2016 and February and June 2017.
ezimmerman@frontlinemedcom.com
On Twitter @eaztweets
, according to new recommendations from the Centers for Disease Control and Prevention.
This change from the CDC’s previous guidance that pregnant women receive a seasonal inactivated influenza vaccine (IIV) was recommended by the Advisory Committee on Immunization Practices after some heated debate among committee members over evidence presented to support the change in wording (MMWR. 2017 Aug 25;66[(RR-2]:1-20).
The new update gives women the ability to choose between receiving an IIV and FluBlok, a recombinant influenza vaccine (RIV) that is not egg based and can be manufactured more quickly, making it ideal in cases of pandemic or supply shortages, according to the CDC.
Although pregnant women may choose to receive a vaccination during the first trimester, the CDC warns there may be some risk involved.
“Although experience with the use of IIVs is substantial, and data from observational studies are available to support the safety of these vaccines in pregnancy, data are more limited for vaccination during the first trimester,” according to the CDC. “Moreover, there is substantially less experience with more recently licensed IIV products (e.g., quadrivalent, cell culture-based, and adjuvanted vaccines) during pregnancy in general.”
Data also are limited regarding RIVs, the CDC said, with the data used to determine safety among pregnant women “limited to reports of pregnancies occurring incidentally during clinical trials, Vaccine Adverse Event Reporting System (VAERS) reports, and pregnancy registry reports.”
Changes for children
The CDC chose to accept ACIP recommendations regarding Afluria (IIV3), expanding the age of children who can receive the vaccine from 9 years and older to 5 years and older.
Similar labeling changes were accepted for FluLaval Quadrivalent (IIV4), which had previously been given to children 3 years and older but now but will be available for children starting at 6 months of age.
New products
Recent product licensures included in the MMWR report are Afluria Quadrivalent (IIV4) and Flublok Quadrivalent (RIV4), both for persons over 18 years.
According to the CDC, Flublok Quadrivalent (an RIV) met noninferiority measures, compared with a similar IIV quadrivalent vaccine, for the A(H3H2) and B/Yamagata viruses but not for A(H1N1) or B/Victoria viruses.
Vaccine composition for 2017-2018
Approved viruses for the 2017-2018 season trivalent vaccines are an A/Michigan/45/2015 (H1N1) pdm09–like virus, an A/Hong Kong/4801/2014 (H3N2)-like virus, and a B/Brisbane/60/2008–like virus (Victoria lineage), according to the MMWR. Quadrivalent vaccines will include those viruses, with the addition of an B/Phuket/3073/2013–like virus (Yamagata lineage).
The CDC continues to recommend that the quadrivalent live attenuated influenza vaccine FluMist not be used by anyone for the 2017-2018 season, a decision that was made after evidence showed poor effectiveness against influenza A(H1N1)pdm09 viruses in the 2013-2014 and 2015-2016 seasons.
Vaccine updates published in this report were recommended by ACIP during meetings held in October 2016 and February and June 2017.
ezimmerman@frontlinemedcom.com
On Twitter @eaztweets
FROM MMWR
FDA grants Priority Review to Gazyva for follicular lymphoma
Gazyva (obinutuzumab) has been granted a Priority Review by the Food and Drug Administration for the treatment of previously untreated follicular lymphoma, according to a press release from Genentech.
FDA approval was based on results from the GALLIUM study, a phase 3 trial comparing Gazyva to Rituxan (rituximab). Patients who received Gazyva plus chemotherapy followed by Gazyva therapy alone for 2 years had a 32% improvement in progression-free survival during the 41.1 month follow-up period, compared with the patient group who received Rituxan plus chemotherapy followed by 2 years of Rituxan therapy alone. Median progression-free survival has not been reached in either arm of the study.
The most common adverse events that occurred more often in the Gazyva arm of the study were low white blood cell count, infections, infusion-related reactions, low platelet count, new tumors, and cardiac events.
“Based on the GALLIUM study, Gazyva-based treatment significantly improved progression-free survival over the current standard of care, and we are committed to bringing this potential new option to patients as soon as possible,” Dr. Sandra Horning, chief medical officer and head of Genentech’s Global Product Development said in the press release.
The FDA is expected to make a decision on approval under Priority Review by Dec. 23, 2017.
Find the full press release on the Genentech website.
Gazyva (obinutuzumab) has been granted a Priority Review by the Food and Drug Administration for the treatment of previously untreated follicular lymphoma, according to a press release from Genentech.
FDA approval was based on results from the GALLIUM study, a phase 3 trial comparing Gazyva to Rituxan (rituximab). Patients who received Gazyva plus chemotherapy followed by Gazyva therapy alone for 2 years had a 32% improvement in progression-free survival during the 41.1 month follow-up period, compared with the patient group who received Rituxan plus chemotherapy followed by 2 years of Rituxan therapy alone. Median progression-free survival has not been reached in either arm of the study.
The most common adverse events that occurred more often in the Gazyva arm of the study were low white blood cell count, infections, infusion-related reactions, low platelet count, new tumors, and cardiac events.
“Based on the GALLIUM study, Gazyva-based treatment significantly improved progression-free survival over the current standard of care, and we are committed to bringing this potential new option to patients as soon as possible,” Dr. Sandra Horning, chief medical officer and head of Genentech’s Global Product Development said in the press release.
The FDA is expected to make a decision on approval under Priority Review by Dec. 23, 2017.
Find the full press release on the Genentech website.
Gazyva (obinutuzumab) has been granted a Priority Review by the Food and Drug Administration for the treatment of previously untreated follicular lymphoma, according to a press release from Genentech.
FDA approval was based on results from the GALLIUM study, a phase 3 trial comparing Gazyva to Rituxan (rituximab). Patients who received Gazyva plus chemotherapy followed by Gazyva therapy alone for 2 years had a 32% improvement in progression-free survival during the 41.1 month follow-up period, compared with the patient group who received Rituxan plus chemotherapy followed by 2 years of Rituxan therapy alone. Median progression-free survival has not been reached in either arm of the study.
The most common adverse events that occurred more often in the Gazyva arm of the study were low white blood cell count, infections, infusion-related reactions, low platelet count, new tumors, and cardiac events.
“Based on the GALLIUM study, Gazyva-based treatment significantly improved progression-free survival over the current standard of care, and we are committed to bringing this potential new option to patients as soon as possible,” Dr. Sandra Horning, chief medical officer and head of Genentech’s Global Product Development said in the press release.
The FDA is expected to make a decision on approval under Priority Review by Dec. 23, 2017.
Find the full press release on the Genentech website.
FDA approves second adalimumab biosimilar for multiple conditions
The Food and Drug Administration has approved Cyltezo (adalimumab-adbm) for multiple conditions.
Cyltezo is an injectable tumor necrosis factor blocker, and is a biosimilar to adalimumab (Humira). The drug is indicated to treat moderate to severe active rheumatoid arthritis, active psoriatic arthritis, active ankylosing spondylitis, moderate to severe active Crohn’s disease, moderate to severe active ulcerative colitis, moderately to severely active polyarticular juvenile idiopathic arthritis in patients 4 years of age and older, and moderate to severe plaque psoriasis.
Find the Cyltezo labeling information here.
The Food and Drug Administration has approved Cyltezo (adalimumab-adbm) for multiple conditions.
Cyltezo is an injectable tumor necrosis factor blocker, and is a biosimilar to adalimumab (Humira). The drug is indicated to treat moderate to severe active rheumatoid arthritis, active psoriatic arthritis, active ankylosing spondylitis, moderate to severe active Crohn’s disease, moderate to severe active ulcerative colitis, moderately to severely active polyarticular juvenile idiopathic arthritis in patients 4 years of age and older, and moderate to severe plaque psoriasis.
Find the Cyltezo labeling information here.
The Food and Drug Administration has approved Cyltezo (adalimumab-adbm) for multiple conditions.
Cyltezo is an injectable tumor necrosis factor blocker, and is a biosimilar to adalimumab (Humira). The drug is indicated to treat moderate to severe active rheumatoid arthritis, active psoriatic arthritis, active ankylosing spondylitis, moderate to severe active Crohn’s disease, moderate to severe active ulcerative colitis, moderately to severely active polyarticular juvenile idiopathic arthritis in patients 4 years of age and older, and moderate to severe plaque psoriasis.
Find the Cyltezo labeling information here.