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FDA Expands Pembrolizumab Approval for Endometrial Cancer
The Food and Drug Administration has expanded the indication for pembrolizumab (Keytruda, Merck) to include the use of the targeted immunotherapy agent plus chemotherapy followed by single-agent pembrolizumab in adults with primary advanced or recurrent endometrial cancer.
Approval in this setting was granted following priority review and was based on efficacy demonstrated in the randomized, placebo-controlled, phase 3 KEYNOTE-868/NRG-GY018 trial. The multicenter trial showed improved progression-free survival (PFS) with chemotherapy plus pembrolizumab versus chemotherapy plus placebo in patients with stage 3 or 4 disease or stage IVB recurrent disease in two cohorts: 222 patients with mismatch repair (MMR) deficiency, and 588 patients with MMR proficiency.
Among the MMR-deficient patients, median PFS was not reached in the treatment arm and was 6.5 months in the control arm (hazard ratio, 0.30). Among the MMR-proficient patients, the median PFS was 11.1 versus 8.5 months in the study arms, respectively (HR, 0.60), according to an FDA press release.
Patients in both cohorts were randomized 1:1 to receive 200 mg of either pembrolizumab or placebo every 3 weeks, followed by paclitaxel at a dose of 175 mg/m2 and carboplatin at a dose of AUC 5 mg/mL/min for six cycles and then 400 mg of pembrolizumab or placebo every 6 weeks for up to 14 cycles.
“Adverse reactions associated with pembrolizumab and chemotherapy were generally similar to those previously reported for pembrolizumab or chemotherapy with the exception of a higher incidence of rash,” the FDA noted.
According to the full prescribing information for pembrolizumab, the recommended dose is 200 mg every 3 weeks or 400 mg every 6 weeks until disease progression, unacceptable toxicity, or for up to 24 months.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration has expanded the indication for pembrolizumab (Keytruda, Merck) to include the use of the targeted immunotherapy agent plus chemotherapy followed by single-agent pembrolizumab in adults with primary advanced or recurrent endometrial cancer.
Approval in this setting was granted following priority review and was based on efficacy demonstrated in the randomized, placebo-controlled, phase 3 KEYNOTE-868/NRG-GY018 trial. The multicenter trial showed improved progression-free survival (PFS) with chemotherapy plus pembrolizumab versus chemotherapy plus placebo in patients with stage 3 or 4 disease or stage IVB recurrent disease in two cohorts: 222 patients with mismatch repair (MMR) deficiency, and 588 patients with MMR proficiency.
Among the MMR-deficient patients, median PFS was not reached in the treatment arm and was 6.5 months in the control arm (hazard ratio, 0.30). Among the MMR-proficient patients, the median PFS was 11.1 versus 8.5 months in the study arms, respectively (HR, 0.60), according to an FDA press release.
Patients in both cohorts were randomized 1:1 to receive 200 mg of either pembrolizumab or placebo every 3 weeks, followed by paclitaxel at a dose of 175 mg/m2 and carboplatin at a dose of AUC 5 mg/mL/min for six cycles and then 400 mg of pembrolizumab or placebo every 6 weeks for up to 14 cycles.
“Adverse reactions associated with pembrolizumab and chemotherapy were generally similar to those previously reported for pembrolizumab or chemotherapy with the exception of a higher incidence of rash,” the FDA noted.
According to the full prescribing information for pembrolizumab, the recommended dose is 200 mg every 3 weeks or 400 mg every 6 weeks until disease progression, unacceptable toxicity, or for up to 24 months.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration has expanded the indication for pembrolizumab (Keytruda, Merck) to include the use of the targeted immunotherapy agent plus chemotherapy followed by single-agent pembrolizumab in adults with primary advanced or recurrent endometrial cancer.
Approval in this setting was granted following priority review and was based on efficacy demonstrated in the randomized, placebo-controlled, phase 3 KEYNOTE-868/NRG-GY018 trial. The multicenter trial showed improved progression-free survival (PFS) with chemotherapy plus pembrolizumab versus chemotherapy plus placebo in patients with stage 3 or 4 disease or stage IVB recurrent disease in two cohorts: 222 patients with mismatch repair (MMR) deficiency, and 588 patients with MMR proficiency.
Among the MMR-deficient patients, median PFS was not reached in the treatment arm and was 6.5 months in the control arm (hazard ratio, 0.30). Among the MMR-proficient patients, the median PFS was 11.1 versus 8.5 months in the study arms, respectively (HR, 0.60), according to an FDA press release.
Patients in both cohorts were randomized 1:1 to receive 200 mg of either pembrolizumab or placebo every 3 weeks, followed by paclitaxel at a dose of 175 mg/m2 and carboplatin at a dose of AUC 5 mg/mL/min for six cycles and then 400 mg of pembrolizumab or placebo every 6 weeks for up to 14 cycles.
“Adverse reactions associated with pembrolizumab and chemotherapy were generally similar to those previously reported for pembrolizumab or chemotherapy with the exception of a higher incidence of rash,” the FDA noted.
According to the full prescribing information for pembrolizumab, the recommended dose is 200 mg every 3 weeks or 400 mg every 6 weeks until disease progression, unacceptable toxicity, or for up to 24 months.
A version of this article first appeared on Medscape.com.
FDA Expands Durvalumab Label to Endometrial Cancer
Originally approved in 2017, the programmed death ligand 1 inhibitor caries previously approved indications for non–small cell lung cancer, biliary tract cancer, and hepatocellular carcinoma.
Approval of the new indication was based on the phase 3 DUO-E trial, which included 95 women with newly diagnosed advanced or recurrent dMMR endometrial cancer. Patients were randomized to durvalumab 1120 mg or placebo with carboplatin plus paclitaxel every 3 weeks for a maximum of six cycles followed by durvalumab 1500 mg every 4 weeks until disease progression.
Median progression-free survival (PFS) was 7 months in the placebo arm but not reached in the durvalumab group. Overall survival outcomes were immature at the PFS analysis.
A quarter or more of durvalumab patients experienced peripheral neuropathy, musculoskeletal pain, nausea, alopecia, fatigue, abdominal pain, constipation, rash, diarrhea, vomiting, and cough.
The recommended treatment regimen for dMMR endometrial cancer in women who weigh ≥ 30 kg is 1120 mg with carboplatin plus paclitaxel every 3 weeks for six cycles, followed by single-agent durvalumab 1500 mg every 4 weeks.
The price of 2.4 mL of durvalumab at a concentration of 50 mg/mL is $1027, according to drugs.com.
A version of this article appeared on Medscape.com.
Originally approved in 2017, the programmed death ligand 1 inhibitor caries previously approved indications for non–small cell lung cancer, biliary tract cancer, and hepatocellular carcinoma.
Approval of the new indication was based on the phase 3 DUO-E trial, which included 95 women with newly diagnosed advanced or recurrent dMMR endometrial cancer. Patients were randomized to durvalumab 1120 mg or placebo with carboplatin plus paclitaxel every 3 weeks for a maximum of six cycles followed by durvalumab 1500 mg every 4 weeks until disease progression.
Median progression-free survival (PFS) was 7 months in the placebo arm but not reached in the durvalumab group. Overall survival outcomes were immature at the PFS analysis.
A quarter or more of durvalumab patients experienced peripheral neuropathy, musculoskeletal pain, nausea, alopecia, fatigue, abdominal pain, constipation, rash, diarrhea, vomiting, and cough.
The recommended treatment regimen for dMMR endometrial cancer in women who weigh ≥ 30 kg is 1120 mg with carboplatin plus paclitaxel every 3 weeks for six cycles, followed by single-agent durvalumab 1500 mg every 4 weeks.
The price of 2.4 mL of durvalumab at a concentration of 50 mg/mL is $1027, according to drugs.com.
A version of this article appeared on Medscape.com.
Originally approved in 2017, the programmed death ligand 1 inhibitor caries previously approved indications for non–small cell lung cancer, biliary tract cancer, and hepatocellular carcinoma.
Approval of the new indication was based on the phase 3 DUO-E trial, which included 95 women with newly diagnosed advanced or recurrent dMMR endometrial cancer. Patients were randomized to durvalumab 1120 mg or placebo with carboplatin plus paclitaxel every 3 weeks for a maximum of six cycles followed by durvalumab 1500 mg every 4 weeks until disease progression.
Median progression-free survival (PFS) was 7 months in the placebo arm but not reached in the durvalumab group. Overall survival outcomes were immature at the PFS analysis.
A quarter or more of durvalumab patients experienced peripheral neuropathy, musculoskeletal pain, nausea, alopecia, fatigue, abdominal pain, constipation, rash, diarrhea, vomiting, and cough.
The recommended treatment regimen for dMMR endometrial cancer in women who weigh ≥ 30 kg is 1120 mg with carboplatin plus paclitaxel every 3 weeks for six cycles, followed by single-agent durvalumab 1500 mg every 4 weeks.
The price of 2.4 mL of durvalumab at a concentration of 50 mg/mL is $1027, according to drugs.com.
A version of this article appeared on Medscape.com.
FDA OKs Iqirvo, First-in-Class PPAR Treatment for Primary Biliary Cholangitis
in combination with ursodeoxycholic acid (UDCA) in adults who do not respond adequately to UDCA or as monotherapy in patients unable to tolerate UDCA.
PBC is a rare, chronic cholestatic liver disease that destroys interlobular bile ducts and leads to cholestasis and liver fibrosis. Left untreated, the disease can worsen over time, leading to cirrhosis and liver transplant and, in some cases, premature death. PBC also harms quality of life, with patients often experiencing severe fatigue and pruritus.
Iqirvo, an oral dual peroxisome proliferator–activated receptor (PPAR) alpha and delta agonist, is the first new drug approved in nearly a decade for treatment of PBC.
Accelerated approval of Iqirvo for PBC was based on data from the phase 3 ELATIVE trial published last year in The New England Journal of Medicine.
The trial randomly assigned patients with PBC who had an inadequate response to or unacceptable side effects with UDCA to receive either once-daily elafibranor (80 mg) or placebo.
The primary endpoint was a biochemical response, defined as an alkaline phosphatase (ALP) level < 1.67 times the upper limit of the normal range, with a reduction ≥ 15% from baseline, as well as normal total bilirubin levels.
Among 161 patients, a biochemical response was seen in 55 of 108 (51%) who received elafibranor vs 2 of 53 (4%) who received placebo.
At week 52, the ALP level normalized in 15% of patients in the elafibranor group and none of the patients in the placebo group.
In a news release announcing approval of Iqirvo, the company notes that improvement in survival and prevention of liver decompensation events have not been demonstrated and that continued approval for PBC may be contingent upon verification and description of clinical benefit in confirmatory trials.
The most common adverse effects with Iqirvo, reported in ≥ 10% of study participants, were weight gain, abdominal pain, diarrhea, nausea, and vomiting. Iqirvo is not recommended for people who have or develop decompensated cirrhosis. Full prescribing information is available online.
The data show that Iqirvo is “an effective second-line treatment for patients with PBC with favorable benefit and risk data,” Kris Kowdley, MD, AGAF, director of the Liver Institute Northwest in Seattle, Washington, and a primary investigator on the ELATIVE study, said in the news release.
The approval of Iqirvo “will allow healthcare providers in the US to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC,” Dr. Kowdley said.
A version of this article appeared on Medscape.com.
in combination with ursodeoxycholic acid (UDCA) in adults who do not respond adequately to UDCA or as monotherapy in patients unable to tolerate UDCA.
PBC is a rare, chronic cholestatic liver disease that destroys interlobular bile ducts and leads to cholestasis and liver fibrosis. Left untreated, the disease can worsen over time, leading to cirrhosis and liver transplant and, in some cases, premature death. PBC also harms quality of life, with patients often experiencing severe fatigue and pruritus.
Iqirvo, an oral dual peroxisome proliferator–activated receptor (PPAR) alpha and delta agonist, is the first new drug approved in nearly a decade for treatment of PBC.
Accelerated approval of Iqirvo for PBC was based on data from the phase 3 ELATIVE trial published last year in The New England Journal of Medicine.
The trial randomly assigned patients with PBC who had an inadequate response to or unacceptable side effects with UDCA to receive either once-daily elafibranor (80 mg) or placebo.
The primary endpoint was a biochemical response, defined as an alkaline phosphatase (ALP) level < 1.67 times the upper limit of the normal range, with a reduction ≥ 15% from baseline, as well as normal total bilirubin levels.
Among 161 patients, a biochemical response was seen in 55 of 108 (51%) who received elafibranor vs 2 of 53 (4%) who received placebo.
At week 52, the ALP level normalized in 15% of patients in the elafibranor group and none of the patients in the placebo group.
In a news release announcing approval of Iqirvo, the company notes that improvement in survival and prevention of liver decompensation events have not been demonstrated and that continued approval for PBC may be contingent upon verification and description of clinical benefit in confirmatory trials.
The most common adverse effects with Iqirvo, reported in ≥ 10% of study participants, were weight gain, abdominal pain, diarrhea, nausea, and vomiting. Iqirvo is not recommended for people who have or develop decompensated cirrhosis. Full prescribing information is available online.
The data show that Iqirvo is “an effective second-line treatment for patients with PBC with favorable benefit and risk data,” Kris Kowdley, MD, AGAF, director of the Liver Institute Northwest in Seattle, Washington, and a primary investigator on the ELATIVE study, said in the news release.
The approval of Iqirvo “will allow healthcare providers in the US to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC,” Dr. Kowdley said.
A version of this article appeared on Medscape.com.
in combination with ursodeoxycholic acid (UDCA) in adults who do not respond adequately to UDCA or as monotherapy in patients unable to tolerate UDCA.
PBC is a rare, chronic cholestatic liver disease that destroys interlobular bile ducts and leads to cholestasis and liver fibrosis. Left untreated, the disease can worsen over time, leading to cirrhosis and liver transplant and, in some cases, premature death. PBC also harms quality of life, with patients often experiencing severe fatigue and pruritus.
Iqirvo, an oral dual peroxisome proliferator–activated receptor (PPAR) alpha and delta agonist, is the first new drug approved in nearly a decade for treatment of PBC.
Accelerated approval of Iqirvo for PBC was based on data from the phase 3 ELATIVE trial published last year in The New England Journal of Medicine.
The trial randomly assigned patients with PBC who had an inadequate response to or unacceptable side effects with UDCA to receive either once-daily elafibranor (80 mg) or placebo.
The primary endpoint was a biochemical response, defined as an alkaline phosphatase (ALP) level < 1.67 times the upper limit of the normal range, with a reduction ≥ 15% from baseline, as well as normal total bilirubin levels.
Among 161 patients, a biochemical response was seen in 55 of 108 (51%) who received elafibranor vs 2 of 53 (4%) who received placebo.
At week 52, the ALP level normalized in 15% of patients in the elafibranor group and none of the patients in the placebo group.
In a news release announcing approval of Iqirvo, the company notes that improvement in survival and prevention of liver decompensation events have not been demonstrated and that continued approval for PBC may be contingent upon verification and description of clinical benefit in confirmatory trials.
The most common adverse effects with Iqirvo, reported in ≥ 10% of study participants, were weight gain, abdominal pain, diarrhea, nausea, and vomiting. Iqirvo is not recommended for people who have or develop decompensated cirrhosis. Full prescribing information is available online.
The data show that Iqirvo is “an effective second-line treatment for patients with PBC with favorable benefit and risk data,” Kris Kowdley, MD, AGAF, director of the Liver Institute Northwest in Seattle, Washington, and a primary investigator on the ELATIVE study, said in the news release.
The approval of Iqirvo “will allow healthcare providers in the US to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC,” Dr. Kowdley said.
A version of this article appeared on Medscape.com.
FDA Expands Repotrectinib Label to All NTRK Gene Fusion+ Solid Tumors
The approval is a label expansion for the tyrosine kinase inhibitor (TKI), which received initial clearance in November 2023 for locally advanced or metastatic ROS1-positive non–small cell lung cancer.
NTRK gene fusions are genetic abnormalities wherein part of the NTRK gene fuses with an unrelated gene. The abnormal gene can then produce an oncogenic protein. Although rare, these mutations are found in many cancer types.
The approval, for adult and pediatric patients aged 12 years or older, was based on the single-arm open-label TRIDENT-1 trial in 88 adults with locally advanced or metastatic NTRK gene fusion solid tumors.
In the 40 patients who were TKI-naive, the overall response rate was 58%, and the median duration of response was not estimable. In the 48 patients who had a TKI previously, the overall response rate was 50% and median duration of response was 9.9 months.
In 20% or more of participants, treatment caused dizziness, dysgeusia, peripheral neuropathy, constipation, dyspnea, fatigue, ataxia, cognitive impairment, muscular weakness, and nausea.
Labeling warns of central nervous system reactions, interstitial lung disease/pneumonitis, hepatotoxicity, myalgia with creatine phosphokinase elevation, hyperuricemia, bone fractures, and embryo-fetal toxicity.
The recommended dose is 160 mg orally once daily for 14 days then increased to 160 mg twice daily until disease progression or unacceptable toxicity.
Sixty 40-mg capsules cost around $7,644, according to drugs.com.
A version of this article appeared on Medscape.com.
The approval is a label expansion for the tyrosine kinase inhibitor (TKI), which received initial clearance in November 2023 for locally advanced or metastatic ROS1-positive non–small cell lung cancer.
NTRK gene fusions are genetic abnormalities wherein part of the NTRK gene fuses with an unrelated gene. The abnormal gene can then produce an oncogenic protein. Although rare, these mutations are found in many cancer types.
The approval, for adult and pediatric patients aged 12 years or older, was based on the single-arm open-label TRIDENT-1 trial in 88 adults with locally advanced or metastatic NTRK gene fusion solid tumors.
In the 40 patients who were TKI-naive, the overall response rate was 58%, and the median duration of response was not estimable. In the 48 patients who had a TKI previously, the overall response rate was 50% and median duration of response was 9.9 months.
In 20% or more of participants, treatment caused dizziness, dysgeusia, peripheral neuropathy, constipation, dyspnea, fatigue, ataxia, cognitive impairment, muscular weakness, and nausea.
Labeling warns of central nervous system reactions, interstitial lung disease/pneumonitis, hepatotoxicity, myalgia with creatine phosphokinase elevation, hyperuricemia, bone fractures, and embryo-fetal toxicity.
The recommended dose is 160 mg orally once daily for 14 days then increased to 160 mg twice daily until disease progression or unacceptable toxicity.
Sixty 40-mg capsules cost around $7,644, according to drugs.com.
A version of this article appeared on Medscape.com.
The approval is a label expansion for the tyrosine kinase inhibitor (TKI), which received initial clearance in November 2023 for locally advanced or metastatic ROS1-positive non–small cell lung cancer.
NTRK gene fusions are genetic abnormalities wherein part of the NTRK gene fuses with an unrelated gene. The abnormal gene can then produce an oncogenic protein. Although rare, these mutations are found in many cancer types.
The approval, for adult and pediatric patients aged 12 years or older, was based on the single-arm open-label TRIDENT-1 trial in 88 adults with locally advanced or metastatic NTRK gene fusion solid tumors.
In the 40 patients who were TKI-naive, the overall response rate was 58%, and the median duration of response was not estimable. In the 48 patients who had a TKI previously, the overall response rate was 50% and median duration of response was 9.9 months.
In 20% or more of participants, treatment caused dizziness, dysgeusia, peripheral neuropathy, constipation, dyspnea, fatigue, ataxia, cognitive impairment, muscular weakness, and nausea.
Labeling warns of central nervous system reactions, interstitial lung disease/pneumonitis, hepatotoxicity, myalgia with creatine phosphokinase elevation, hyperuricemia, bone fractures, and embryo-fetal toxicity.
The recommended dose is 160 mg orally once daily for 14 days then increased to 160 mg twice daily until disease progression or unacceptable toxicity.
Sixty 40-mg capsules cost around $7,644, according to drugs.com.
A version of this article appeared on Medscape.com.
FDA Approves Tarlatamab for Extensive-Stage Small Cell Lung Cancer
Tarlatamab is a first-in-class bispecific T-cell engager (BiTE) that binds delta-like ligand 3 on the surface of cells, including tumor cells, and CD3 expressed on the surface of T cells. It causes T-cell activation, release of inflammatory cytokines, and lysis of DLL3-expressing cells, according to labeling.
Approval was based on data from 99 patients in the DeLLphi-301 trial with relapsed/refractory extensive-stage SCLC who had progressed after platinum-based chemotherapy. Patients with symptomatic brain metastases, interstitial lung disease, noninfectious pneumonitis, and active immunodeficiency were excluded.
The overall response rate was 40%, and median duration of response 9.7 months. The overall response rate was 52% in 27 patients with platinum-resistant SCLC and 31% in 42 with platinum-sensitive disease.
Continued approval may depend on verification of clinical benefit in a confirmatory trial.
Labeling includes a box warning of serious or life-threatening cytokine release syndrome and neurologic toxicity, including immune effector cell–associated neurotoxicity syndrome.
The most common adverse events, occurring in 20% or more of patients, were cytokine release syndrome, fatigue, pyrexia, dysgeusia, decreased appetite, musculoskeletal pain, constipation, anemia, and nausea.
The most common grade 3 or 4 laboratory abnormalities included decreased lymphocytes, decreased sodium, increased uric acid, decreased total neutrophils, decreased hemoglobin, increased activated partial thromboplastin time, and decreased potassium.
The starting dose is 1 mg given intravenously over 1 hour on the first day of the first cycle followed by 10 mg on day 8 and day 15 of the first cycle, then every 2 weeks until disease progression or unacceptable toxicity.
M. Alexander Otto is a physician assistant with a master’s degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: [email protected]
A version of this article appeared on Medscape.com.
Tarlatamab is a first-in-class bispecific T-cell engager (BiTE) that binds delta-like ligand 3 on the surface of cells, including tumor cells, and CD3 expressed on the surface of T cells. It causes T-cell activation, release of inflammatory cytokines, and lysis of DLL3-expressing cells, according to labeling.
Approval was based on data from 99 patients in the DeLLphi-301 trial with relapsed/refractory extensive-stage SCLC who had progressed after platinum-based chemotherapy. Patients with symptomatic brain metastases, interstitial lung disease, noninfectious pneumonitis, and active immunodeficiency were excluded.
The overall response rate was 40%, and median duration of response 9.7 months. The overall response rate was 52% in 27 patients with platinum-resistant SCLC and 31% in 42 with platinum-sensitive disease.
Continued approval may depend on verification of clinical benefit in a confirmatory trial.
Labeling includes a box warning of serious or life-threatening cytokine release syndrome and neurologic toxicity, including immune effector cell–associated neurotoxicity syndrome.
The most common adverse events, occurring in 20% or more of patients, were cytokine release syndrome, fatigue, pyrexia, dysgeusia, decreased appetite, musculoskeletal pain, constipation, anemia, and nausea.
The most common grade 3 or 4 laboratory abnormalities included decreased lymphocytes, decreased sodium, increased uric acid, decreased total neutrophils, decreased hemoglobin, increased activated partial thromboplastin time, and decreased potassium.
The starting dose is 1 mg given intravenously over 1 hour on the first day of the first cycle followed by 10 mg on day 8 and day 15 of the first cycle, then every 2 weeks until disease progression or unacceptable toxicity.
M. Alexander Otto is a physician assistant with a master’s degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: [email protected]
A version of this article appeared on Medscape.com.
Tarlatamab is a first-in-class bispecific T-cell engager (BiTE) that binds delta-like ligand 3 on the surface of cells, including tumor cells, and CD3 expressed on the surface of T cells. It causes T-cell activation, release of inflammatory cytokines, and lysis of DLL3-expressing cells, according to labeling.
Approval was based on data from 99 patients in the DeLLphi-301 trial with relapsed/refractory extensive-stage SCLC who had progressed after platinum-based chemotherapy. Patients with symptomatic brain metastases, interstitial lung disease, noninfectious pneumonitis, and active immunodeficiency were excluded.
The overall response rate was 40%, and median duration of response 9.7 months. The overall response rate was 52% in 27 patients with platinum-resistant SCLC and 31% in 42 with platinum-sensitive disease.
Continued approval may depend on verification of clinical benefit in a confirmatory trial.
Labeling includes a box warning of serious or life-threatening cytokine release syndrome and neurologic toxicity, including immune effector cell–associated neurotoxicity syndrome.
The most common adverse events, occurring in 20% or more of patients, were cytokine release syndrome, fatigue, pyrexia, dysgeusia, decreased appetite, musculoskeletal pain, constipation, anemia, and nausea.
The most common grade 3 or 4 laboratory abnormalities included decreased lymphocytes, decreased sodium, increased uric acid, decreased total neutrophils, decreased hemoglobin, increased activated partial thromboplastin time, and decreased potassium.
The starting dose is 1 mg given intravenously over 1 hour on the first day of the first cycle followed by 10 mg on day 8 and day 15 of the first cycle, then every 2 weeks until disease progression or unacceptable toxicity.
M. Alexander Otto is a physician assistant with a master’s degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: [email protected]
A version of this article appeared on Medscape.com.
FDA Broadens Breyanzi’s Follicular Lymphoma Indication
The approval broadens the use of the CAR T-cell therapy for follicular lymphoma. Previous approval was limited to relapsed/refractory grade 3B disease. Lisocabtagene maraleucel also carries relapsed/refractory B-cell lymphoma and lymphocytic leukemia indications.
The new approval was based on the phase 2 single-arm TRANSCEND FL trial in 94 patients with relapsed/refractory follicular lymphoma after two or more lines of systemic therapy, including an anti-CD20 antibody and an alkylating agent.
Adequate bone marrow function and a performance score of 0-1 were required.
Patients received a single dose 2-7 days after completing lymphodepleting chemotherapy.
The overall response rate was 95.7%. The median duration of response was not reached after a median follow-up of 16.8 months.
The most common nonlaboratory adverse events, occurring in at least 20% of patients, were cytokine release syndrome, headache, musculoskeletal pain, fatigue, constipation, and fever.
Lisocabtagene maraleucel is available only through a Risk Evaluation and Mitigation Strategy program due to the risk for fatal cytokine release syndrome and neurologic toxicities.
A single treatment is almost a half million dollars, according to drugs.com.
A version of this article appeared on Medscape.com.
The approval broadens the use of the CAR T-cell therapy for follicular lymphoma. Previous approval was limited to relapsed/refractory grade 3B disease. Lisocabtagene maraleucel also carries relapsed/refractory B-cell lymphoma and lymphocytic leukemia indications.
The new approval was based on the phase 2 single-arm TRANSCEND FL trial in 94 patients with relapsed/refractory follicular lymphoma after two or more lines of systemic therapy, including an anti-CD20 antibody and an alkylating agent.
Adequate bone marrow function and a performance score of 0-1 were required.
Patients received a single dose 2-7 days after completing lymphodepleting chemotherapy.
The overall response rate was 95.7%. The median duration of response was not reached after a median follow-up of 16.8 months.
The most common nonlaboratory adverse events, occurring in at least 20% of patients, were cytokine release syndrome, headache, musculoskeletal pain, fatigue, constipation, and fever.
Lisocabtagene maraleucel is available only through a Risk Evaluation and Mitigation Strategy program due to the risk for fatal cytokine release syndrome and neurologic toxicities.
A single treatment is almost a half million dollars, according to drugs.com.
A version of this article appeared on Medscape.com.
The approval broadens the use of the CAR T-cell therapy for follicular lymphoma. Previous approval was limited to relapsed/refractory grade 3B disease. Lisocabtagene maraleucel also carries relapsed/refractory B-cell lymphoma and lymphocytic leukemia indications.
The new approval was based on the phase 2 single-arm TRANSCEND FL trial in 94 patients with relapsed/refractory follicular lymphoma after two or more lines of systemic therapy, including an anti-CD20 antibody and an alkylating agent.
Adequate bone marrow function and a performance score of 0-1 were required.
Patients received a single dose 2-7 days after completing lymphodepleting chemotherapy.
The overall response rate was 95.7%. The median duration of response was not reached after a median follow-up of 16.8 months.
The most common nonlaboratory adverse events, occurring in at least 20% of patients, were cytokine release syndrome, headache, musculoskeletal pain, fatigue, constipation, and fever.
Lisocabtagene maraleucel is available only through a Risk Evaluation and Mitigation Strategy program due to the risk for fatal cytokine release syndrome and neurologic toxicities.
A single treatment is almost a half million dollars, according to drugs.com.
A version of this article appeared on Medscape.com.
OTC Solution for Erectile Dysfunction?
Up to 60% of men with erectile dysfunction who were not candidates for phosphodiesterase 5 (PDE5) inhibitors achieved erections in less than 10 minutes after a single application of a first-on-the-market nonprescription gel to the glans, a new study found.
Wayne Hellstrom, MD, chief of andrology at Tulane School of Medicine in New Orleans, who presented the study of MED3000 [Eroxon] on May 5 at the 2024 annual meeting of the American Urological Association in San Antonio, Texas, said that the gel is considered to be a device by the US Food and Drug Administration (FDA). The agency approved the product in June 2023.
A spokesman for Futura, which makes MED3000, said that the gel will be on the market 2025. No price for the United States has been announced, but a four-pack of single-use tubes sells for the equivalent of roughly $31 in the United Kingdom.
Dr. Hellstrom, a former adviser to Futura, he said he expects MED3000 will be “a potential first-line therapy in addition to PDE5 inhibitors,” which are vasodilating drugs that stimulate the corpora cavernosa of the penis, facilitating erection with sexual stimulation.
He noted that PDE5s are contraindicated for many men; are not tolerated in others; are not completely effective; or work too slowly, taking 1-2 hours to work. As a result, up to 50% of patients cease using a PDE5 inhibitor within 1 year, he said.
Futura said the gel contains a combination of volatile solvents which, when applied to the head of the penis, evaporate rapidly, stimulating nerve endings through an initial cooling effect followed by a warming sensation. This reaction releases nitric oxide, relaxing the smooth muscle tissue inside the penis and increasing blood flow that is needed to obtain an erection.
Dr. Hellstrom noted that MED3000 is noninvasive and causes no side effects and is slightly more effective if applied by a partner.
The new findings come from two studies of 250 men with erectile dysfunction (FM57) who used MED3000 over 12 weeks and a randomly assigned arm (FM71) with two groups of 48 men who used either MED3000 or 5 mg of tadalafil over 24 weeks.
Erections were achieved in less than 10 minutes in 60.1% of men in the FM57 group and 44.9% of those in the FM71 group.
Overall, less than 2% of the men who usedMED3000 and 4% of those who took tadalafil reported adverse effects. These events included headaches in 3% of the combined MED3000 group and 19.1% of the tadalafil group. Roughly 1% of men who used MED3000 reported penile burning sensation compared with none in the group taking tadalafil.
Problematic Design?
Kevin McVary, MD, a professor of urology at Stritch School of Medicine of Loyola University, outside of Chicago, and director of the Center for Male Health, criticized the study design and added that he did not believe MED3000 had been proven beneficial.
“Are they expecting the Cialis 5 mg to work within 10 minutes? Because it doesn’t,” Dr. McVary said. “It doesn’t get absorbed into the bloodstream for about 2.5 hours.”
Dr. McVary said that men with erectile dysfunction will probably do anything to avoid seeing a physician about the condition, which could make MED3000 highly marketable.
However, he said, examinations would be important to detect unrecognized underlying cardiac disease, especially in younger men. “ED can function as the classic canary in a coal mine where it tells you who’s at risk for unexpected early death,” he said.
Dr. Hellstrom is a former adviser to Futura Medical Developments, which funded the research. Dr. McVary reported no relevant financial conflicts of interest.
A version of this article appeared on Medscape.com .
Up to 60% of men with erectile dysfunction who were not candidates for phosphodiesterase 5 (PDE5) inhibitors achieved erections in less than 10 minutes after a single application of a first-on-the-market nonprescription gel to the glans, a new study found.
Wayne Hellstrom, MD, chief of andrology at Tulane School of Medicine in New Orleans, who presented the study of MED3000 [Eroxon] on May 5 at the 2024 annual meeting of the American Urological Association in San Antonio, Texas, said that the gel is considered to be a device by the US Food and Drug Administration (FDA). The agency approved the product in June 2023.
A spokesman for Futura, which makes MED3000, said that the gel will be on the market 2025. No price for the United States has been announced, but a four-pack of single-use tubes sells for the equivalent of roughly $31 in the United Kingdom.
Dr. Hellstrom, a former adviser to Futura, he said he expects MED3000 will be “a potential first-line therapy in addition to PDE5 inhibitors,” which are vasodilating drugs that stimulate the corpora cavernosa of the penis, facilitating erection with sexual stimulation.
He noted that PDE5s are contraindicated for many men; are not tolerated in others; are not completely effective; or work too slowly, taking 1-2 hours to work. As a result, up to 50% of patients cease using a PDE5 inhibitor within 1 year, he said.
Futura said the gel contains a combination of volatile solvents which, when applied to the head of the penis, evaporate rapidly, stimulating nerve endings through an initial cooling effect followed by a warming sensation. This reaction releases nitric oxide, relaxing the smooth muscle tissue inside the penis and increasing blood flow that is needed to obtain an erection.
Dr. Hellstrom noted that MED3000 is noninvasive and causes no side effects and is slightly more effective if applied by a partner.
The new findings come from two studies of 250 men with erectile dysfunction (FM57) who used MED3000 over 12 weeks and a randomly assigned arm (FM71) with two groups of 48 men who used either MED3000 or 5 mg of tadalafil over 24 weeks.
Erections were achieved in less than 10 minutes in 60.1% of men in the FM57 group and 44.9% of those in the FM71 group.
Overall, less than 2% of the men who usedMED3000 and 4% of those who took tadalafil reported adverse effects. These events included headaches in 3% of the combined MED3000 group and 19.1% of the tadalafil group. Roughly 1% of men who used MED3000 reported penile burning sensation compared with none in the group taking tadalafil.
Problematic Design?
Kevin McVary, MD, a professor of urology at Stritch School of Medicine of Loyola University, outside of Chicago, and director of the Center for Male Health, criticized the study design and added that he did not believe MED3000 had been proven beneficial.
“Are they expecting the Cialis 5 mg to work within 10 minutes? Because it doesn’t,” Dr. McVary said. “It doesn’t get absorbed into the bloodstream for about 2.5 hours.”
Dr. McVary said that men with erectile dysfunction will probably do anything to avoid seeing a physician about the condition, which could make MED3000 highly marketable.
However, he said, examinations would be important to detect unrecognized underlying cardiac disease, especially in younger men. “ED can function as the classic canary in a coal mine where it tells you who’s at risk for unexpected early death,” he said.
Dr. Hellstrom is a former adviser to Futura Medical Developments, which funded the research. Dr. McVary reported no relevant financial conflicts of interest.
A version of this article appeared on Medscape.com .
Up to 60% of men with erectile dysfunction who were not candidates for phosphodiesterase 5 (PDE5) inhibitors achieved erections in less than 10 minutes after a single application of a first-on-the-market nonprescription gel to the glans, a new study found.
Wayne Hellstrom, MD, chief of andrology at Tulane School of Medicine in New Orleans, who presented the study of MED3000 [Eroxon] on May 5 at the 2024 annual meeting of the American Urological Association in San Antonio, Texas, said that the gel is considered to be a device by the US Food and Drug Administration (FDA). The agency approved the product in June 2023.
A spokesman for Futura, which makes MED3000, said that the gel will be on the market 2025. No price for the United States has been announced, but a four-pack of single-use tubes sells for the equivalent of roughly $31 in the United Kingdom.
Dr. Hellstrom, a former adviser to Futura, he said he expects MED3000 will be “a potential first-line therapy in addition to PDE5 inhibitors,” which are vasodilating drugs that stimulate the corpora cavernosa of the penis, facilitating erection with sexual stimulation.
He noted that PDE5s are contraindicated for many men; are not tolerated in others; are not completely effective; or work too slowly, taking 1-2 hours to work. As a result, up to 50% of patients cease using a PDE5 inhibitor within 1 year, he said.
Futura said the gel contains a combination of volatile solvents which, when applied to the head of the penis, evaporate rapidly, stimulating nerve endings through an initial cooling effect followed by a warming sensation. This reaction releases nitric oxide, relaxing the smooth muscle tissue inside the penis and increasing blood flow that is needed to obtain an erection.
Dr. Hellstrom noted that MED3000 is noninvasive and causes no side effects and is slightly more effective if applied by a partner.
The new findings come from two studies of 250 men with erectile dysfunction (FM57) who used MED3000 over 12 weeks and a randomly assigned arm (FM71) with two groups of 48 men who used either MED3000 or 5 mg of tadalafil over 24 weeks.
Erections were achieved in less than 10 minutes in 60.1% of men in the FM57 group and 44.9% of those in the FM71 group.
Overall, less than 2% of the men who usedMED3000 and 4% of those who took tadalafil reported adverse effects. These events included headaches in 3% of the combined MED3000 group and 19.1% of the tadalafil group. Roughly 1% of men who used MED3000 reported penile burning sensation compared with none in the group taking tadalafil.
Problematic Design?
Kevin McVary, MD, a professor of urology at Stritch School of Medicine of Loyola University, outside of Chicago, and director of the Center for Male Health, criticized the study design and added that he did not believe MED3000 had been proven beneficial.
“Are they expecting the Cialis 5 mg to work within 10 minutes? Because it doesn’t,” Dr. McVary said. “It doesn’t get absorbed into the bloodstream for about 2.5 hours.”
Dr. McVary said that men with erectile dysfunction will probably do anything to avoid seeing a physician about the condition, which could make MED3000 highly marketable.
However, he said, examinations would be important to detect unrecognized underlying cardiac disease, especially in younger men. “ED can function as the classic canary in a coal mine where it tells you who’s at risk for unexpected early death,” he said.
Dr. Hellstrom is a former adviser to Futura Medical Developments, which funded the research. Dr. McVary reported no relevant financial conflicts of interest.
A version of this article appeared on Medscape.com .
FDA Allows Implantable CGM to Integrate With Insulin Pumps
The US Food and Drug Administration (FDA) has designated the Eversense (Sensionics, Inc; Ascencia Diabetes Care) implanted continuous glucose monitor (CGM) an “integrated CGM,” meaning it can be used in conjunction with insulin pumps as part of an automated insulin delivery system (AID).
The Eversense now joins Dexcom’s G6 and G7 and the Freestyle Libre 2 Plus in being compatible with multiple different branded insulin pumps as part of AID systems, and it is the only implantable one.
The sensor device is inserted under the skin of the patient’s upper arm by a healthcare provider and a transmitter is worn over it on the skin. The FDA approved the Eversense in June 2018 for 3-month use and in February 2022 for use up to 6 months. It is indicated for people with diabetes aged 18 years and older.
Fingerstick blood glucose measurements are still required for calibration once a day after day 21, when symptoms don’t match the CGM information, or when taking tetracycline medications.
According to Sensionics, the Eversense is “the most accurate CGM in the critical low glucose ranges with essentially no compression lows.” The latter refers to ‘false low’ alarms that sometimes occur when a person presses on the device, such as during sleep.
“As we look ahead, we are focused on progressing our partnership discussions and software developments, and look forward to providing more updates,” Sensionics said in a statement.
A version of this article first appeared on Medscape.com.
The US Food and Drug Administration (FDA) has designated the Eversense (Sensionics, Inc; Ascencia Diabetes Care) implanted continuous glucose monitor (CGM) an “integrated CGM,” meaning it can be used in conjunction with insulin pumps as part of an automated insulin delivery system (AID).
The Eversense now joins Dexcom’s G6 and G7 and the Freestyle Libre 2 Plus in being compatible with multiple different branded insulin pumps as part of AID systems, and it is the only implantable one.
The sensor device is inserted under the skin of the patient’s upper arm by a healthcare provider and a transmitter is worn over it on the skin. The FDA approved the Eversense in June 2018 for 3-month use and in February 2022 for use up to 6 months. It is indicated for people with diabetes aged 18 years and older.
Fingerstick blood glucose measurements are still required for calibration once a day after day 21, when symptoms don’t match the CGM information, or when taking tetracycline medications.
According to Sensionics, the Eversense is “the most accurate CGM in the critical low glucose ranges with essentially no compression lows.” The latter refers to ‘false low’ alarms that sometimes occur when a person presses on the device, such as during sleep.
“As we look ahead, we are focused on progressing our partnership discussions and software developments, and look forward to providing more updates,” Sensionics said in a statement.
A version of this article first appeared on Medscape.com.
The US Food and Drug Administration (FDA) has designated the Eversense (Sensionics, Inc; Ascencia Diabetes Care) implanted continuous glucose monitor (CGM) an “integrated CGM,” meaning it can be used in conjunction with insulin pumps as part of an automated insulin delivery system (AID).
The Eversense now joins Dexcom’s G6 and G7 and the Freestyle Libre 2 Plus in being compatible with multiple different branded insulin pumps as part of AID systems, and it is the only implantable one.
The sensor device is inserted under the skin of the patient’s upper arm by a healthcare provider and a transmitter is worn over it on the skin. The FDA approved the Eversense in June 2018 for 3-month use and in February 2022 for use up to 6 months. It is indicated for people with diabetes aged 18 years and older.
Fingerstick blood glucose measurements are still required for calibration once a day after day 21, when symptoms don’t match the CGM information, or when taking tetracycline medications.
According to Sensionics, the Eversense is “the most accurate CGM in the critical low glucose ranges with essentially no compression lows.” The latter refers to ‘false low’ alarms that sometimes occur when a person presses on the device, such as during sleep.
“As we look ahead, we are focused on progressing our partnership discussions and software developments, and look forward to providing more updates,” Sensionics said in a statement.
A version of this article first appeared on Medscape.com.
FDA Approves New Antibiotic for Uncomplicated UTIs
The US Food and Drug Administration (FDA) has approved a new treatment for uncomplicated urinary tract infections (UTIs).
The agency on April 24 approved pivmecillinam tablets to treat women aged 18 years or older with UTIs caused by bacteria susceptible to the drug.
The beta-lactam antibiotic already is approved in Europe and has been used for more than 40 years outside of the United States to treat infections, according to the drug’s manufacturer, Utility Therapeutics.
The drug is an aminopenicillin that rapidly converts to mecillinam, according to the company, which is marketing the medication as Pivya.
Pivmecillinam is intended to treat UTIs caused by susceptible isolates of Escherichia coli, Proteus mirabilis, and Staphylococcus saprophyticus.
Researchers studied the treatment in three clinical trials. One study found women who received the new antibiotic were more likely to have resolution of symptoms and a reduction in bacteria in urine compared with placebo (62% vs 10%). Similar results were seen in a trial that used ibuprofen as the comparator (66% vs 22%).
In a third study that assessed two oral antibacterial drugs, 72% of women who received pivmecillinam and 76% who received the other drug achieved resolution of symptoms and a reduction in bacteria, according to the FDA.
The most common side effects of pivmecillinam include nausea and diarrhea.
About half of all women will experience at least one UTI in their lifetime, and the infections are one the top reasons for antibiotic prescriptions, the FDA noted.
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved a new treatment for uncomplicated urinary tract infections (UTIs).
The agency on April 24 approved pivmecillinam tablets to treat women aged 18 years or older with UTIs caused by bacteria susceptible to the drug.
The beta-lactam antibiotic already is approved in Europe and has been used for more than 40 years outside of the United States to treat infections, according to the drug’s manufacturer, Utility Therapeutics.
The drug is an aminopenicillin that rapidly converts to mecillinam, according to the company, which is marketing the medication as Pivya.
Pivmecillinam is intended to treat UTIs caused by susceptible isolates of Escherichia coli, Proteus mirabilis, and Staphylococcus saprophyticus.
Researchers studied the treatment in three clinical trials. One study found women who received the new antibiotic were more likely to have resolution of symptoms and a reduction in bacteria in urine compared with placebo (62% vs 10%). Similar results were seen in a trial that used ibuprofen as the comparator (66% vs 22%).
In a third study that assessed two oral antibacterial drugs, 72% of women who received pivmecillinam and 76% who received the other drug achieved resolution of symptoms and a reduction in bacteria, according to the FDA.
The most common side effects of pivmecillinam include nausea and diarrhea.
About half of all women will experience at least one UTI in their lifetime, and the infections are one the top reasons for antibiotic prescriptions, the FDA noted.
A version of this article appeared on Medscape.com.
The US Food and Drug Administration (FDA) has approved a new treatment for uncomplicated urinary tract infections (UTIs).
The agency on April 24 approved pivmecillinam tablets to treat women aged 18 years or older with UTIs caused by bacteria susceptible to the drug.
The beta-lactam antibiotic already is approved in Europe and has been used for more than 40 years outside of the United States to treat infections, according to the drug’s manufacturer, Utility Therapeutics.
The drug is an aminopenicillin that rapidly converts to mecillinam, according to the company, which is marketing the medication as Pivya.
Pivmecillinam is intended to treat UTIs caused by susceptible isolates of Escherichia coli, Proteus mirabilis, and Staphylococcus saprophyticus.
Researchers studied the treatment in three clinical trials. One study found women who received the new antibiotic were more likely to have resolution of symptoms and a reduction in bacteria in urine compared with placebo (62% vs 10%). Similar results were seen in a trial that used ibuprofen as the comparator (66% vs 22%).
In a third study that assessed two oral antibacterial drugs, 72% of women who received pivmecillinam and 76% who received the other drug achieved resolution of symptoms and a reduction in bacteria, according to the FDA.
The most common side effects of pivmecillinam include nausea and diarrhea.
About half of all women will experience at least one UTI in their lifetime, and the infections are one the top reasons for antibiotic prescriptions, the FDA noted.
A version of this article appeared on Medscape.com.
FDA Requests More Information for RDEB Rx Under Review
The Food and Drug Administration (RDEB), requesting more information from the manufacturer.
Pz-cel, which comprises autologous, COL7A1 gene–corrected epidermal sheets, is being evaluated for its ability to enable normal type VII collagen expression in a patient’s skin cells and to facilitate wound healing and pain reduction in wounds in patients with RDEB after a one-time application procedure. The cause of RDEB is a defect in the COL7A1 gene that “results in the inability to produce type VII collagen,” a press release from the manufacturer noted.
On April 22, 2024, the manufacturer Abeona Therapeutics announced that following a meeting with the FDA in March and in a subsequent request for information, the agency requires additional information to satisfy certain Chemistry Manufacturing and Controls requirements before the BLA for pz-cel can be approved. According to a press release from the company, the information pertains to validation requirements for certain manufacturing and release testing methods, including some that were observed during the FDA’s pre-licensing inspection.
The complete response letter did not identify any issues related to the clinical efficacy or safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support approval, according to the company.
The company anticipates completing the BLA resubmission in the third quarter of 2024. The application is supported by clinical efficacy and safety data from the pivotal phase 3 VIITAL study and a phase 1/2a study in patients with RDEB.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration (RDEB), requesting more information from the manufacturer.
Pz-cel, which comprises autologous, COL7A1 gene–corrected epidermal sheets, is being evaluated for its ability to enable normal type VII collagen expression in a patient’s skin cells and to facilitate wound healing and pain reduction in wounds in patients with RDEB after a one-time application procedure. The cause of RDEB is a defect in the COL7A1 gene that “results in the inability to produce type VII collagen,” a press release from the manufacturer noted.
On April 22, 2024, the manufacturer Abeona Therapeutics announced that following a meeting with the FDA in March and in a subsequent request for information, the agency requires additional information to satisfy certain Chemistry Manufacturing and Controls requirements before the BLA for pz-cel can be approved. According to a press release from the company, the information pertains to validation requirements for certain manufacturing and release testing methods, including some that were observed during the FDA’s pre-licensing inspection.
The complete response letter did not identify any issues related to the clinical efficacy or safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support approval, according to the company.
The company anticipates completing the BLA resubmission in the third quarter of 2024. The application is supported by clinical efficacy and safety data from the pivotal phase 3 VIITAL study and a phase 1/2a study in patients with RDEB.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration (RDEB), requesting more information from the manufacturer.
Pz-cel, which comprises autologous, COL7A1 gene–corrected epidermal sheets, is being evaluated for its ability to enable normal type VII collagen expression in a patient’s skin cells and to facilitate wound healing and pain reduction in wounds in patients with RDEB after a one-time application procedure. The cause of RDEB is a defect in the COL7A1 gene that “results in the inability to produce type VII collagen,” a press release from the manufacturer noted.
On April 22, 2024, the manufacturer Abeona Therapeutics announced that following a meeting with the FDA in March and in a subsequent request for information, the agency requires additional information to satisfy certain Chemistry Manufacturing and Controls requirements before the BLA for pz-cel can be approved. According to a press release from the company, the information pertains to validation requirements for certain manufacturing and release testing methods, including some that were observed during the FDA’s pre-licensing inspection.
The complete response letter did not identify any issues related to the clinical efficacy or safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support approval, according to the company.
The company anticipates completing the BLA resubmission in the third quarter of 2024. The application is supported by clinical efficacy and safety data from the pivotal phase 3 VIITAL study and a phase 1/2a study in patients with RDEB.
A version of this article first appeared on Medscape.com.