FDA/CDC

The US Food and Drug Administration granted accelerated approval to epcoritamab-bysp (Epkinly, Genmab US) for adults with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

This marks the second indication for the bispecific CD20-directed CD3 T-cell engager. The agent was first approved in 2023 for relapsed or refractory diffuse large B-cell lymphoma in adults.

The current approval was based on the single-arm EPCORE NHL-1 trial in 127 patients with follicular lymphoma who had received at least two lines of systemic therapy.

After a two step-up dosing regimen, the overall response rate was 82%, with 60% of patients achieving a complete response. At a median follow-up of 14.8 months, the median duration of response was not reached. The 12-month duration of response was 68.4%.

Efficacy was similar in the 86 patients who received a three step-up dosing schedule.

Labeling carries a black box warning of cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome. Adverse events in 20% or more of patients included injection site reactions, cytokine release syndrome, COVID-19 infection, fatigue, upper respiratory tract infection, musculoskeletal pain, rash, diarrhea, pyrexia, cough, and headache.

Decreased lymphocyte count, neutrophil count, white blood cell count, and hemoglobin were the most common grade 3/4 laboratory abnormalities.

Three step-up dosing is the recommended regimen, with epcoritamab administered subcutaneously in 28-day cycles until disease progression or unacceptable toxicity. Dosing is increased by steps to the full 48 mg in cycle 1.

The price is $16,282.52 for 48 mg/0.8 mL, according to drugs.com.

A version of this article appeared on Medscape.com.

The US Food and Drug Administration granted accelerated approval to epcoritamab-bysp (Epkinly, Genmab US) for adults with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

This marks the second indication for the bispecific CD20-directed CD3 T-cell engager. The agent was first approved in 2023 for relapsed or refractory diffuse large B-cell lymphoma in adults.

The current approval was based on the single-arm EPCORE NHL-1 trial in 127 patients with follicular lymphoma who had received at least two lines of systemic therapy.

After a two step-up dosing regimen, the overall response rate was 82%, with 60% of patients achieving a complete response. At a median follow-up of 14.8 months, the median duration of response was not reached. The 12-month duration of response was 68.4%.

Efficacy was similar in the 86 patients who received a three step-up dosing schedule.

Labeling carries a black box warning of cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome. Adverse events in 20% or more of patients included injection site reactions, cytokine release syndrome, COVID-19 infection, fatigue, upper respiratory tract infection, musculoskeletal pain, rash, diarrhea, pyrexia, cough, and headache.

Decreased lymphocyte count, neutrophil count, white blood cell count, and hemoglobin were the most common grade 3/4 laboratory abnormalities.

Three step-up dosing is the recommended regimen, with epcoritamab administered subcutaneously in 28-day cycles until disease progression or unacceptable toxicity. Dosing is increased by steps to the full 48 mg in cycle 1.

The price is $16,282.52 for 48 mg/0.8 mL, according to drugs.com.

A version of this article appeared on Medscape.com.

The US Food and Drug Administration granted accelerated approval to epcoritamab-bysp (Epkinly, Genmab US) for adults with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

This marks the second indication for the bispecific CD20-directed CD3 T-cell engager. The agent was first approved in 2023 for relapsed or refractory diffuse large B-cell lymphoma in adults.

The current approval was based on the single-arm EPCORE NHL-1 trial in 127 patients with follicular lymphoma who had received at least two lines of systemic therapy.

After a two step-up dosing regimen, the overall response rate was 82%, with 60% of patients achieving a complete response. At a median follow-up of 14.8 months, the median duration of response was not reached. The 12-month duration of response was 68.4%.

Efficacy was similar in the 86 patients who received a three step-up dosing schedule.

Labeling carries a black box warning of cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome. Adverse events in 20% or more of patients included injection site reactions, cytokine release syndrome, COVID-19 infection, fatigue, upper respiratory tract infection, musculoskeletal pain, rash, diarrhea, pyrexia, cough, and headache.

Decreased lymphocyte count, neutrophil count, white blood cell count, and hemoglobin were the most common grade 3/4 laboratory abnormalities.

Three step-up dosing is the recommended regimen, with epcoritamab administered subcutaneously in 28-day cycles until disease progression or unacceptable toxicity. Dosing is increased by steps to the full 48 mg in cycle 1.

The price is $16,282.52 for 48 mg/0.8 mL, according to drugs.com.

A version of this article appeared on Medscape.com.

The US Food and Drug Administration (FDA) has granted accelerated approval to adagrasib (Krazati, Mirati Therapeutics) with cetuximab for certain patients with KRAS G12C–mutated colorectal cancer (CRC).

More specifically, the highly selective and potent small-molecule KRAS G12C inhibitor is now indicated for patients with locally advanced or metastatic KRAS G12C–mutated CRC — as determined by an FDA-approved test — who previously received fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy and, if eligible, a vascular endothelial growth factor inhibitor, according to an FDA press release.

The agent is the first KRAS inhibitor approved for CRC. Adagrasib was previously granted accelerated approval for KRAS G12C–mutated non–small cell lung cancer, based on findings from the KRYSTAL-12 trial.

The CRC approval was based on findings from the KRYSTAL-1 multicenter, single-arm expansion cohort trial, which reported an overall response rate of 34% among 94 enrolled patients. 

All responses were partial responses, and the median duration of response was 5.8 months, with 31% of responding patients experiencing a duration of response of at least 6 months.

Patients received 600 mg of adagrasib twice daily plus cetuximab administered in either a biweekly 500 mg/m² dose or an initial dose of 400 mg/m² followed by weekly doses of 250 mg/m². Those who discontinued adagrasib also had to discontinue cetuximab, but adagrasib could be continued if cetuximab was discontinued.

The recommended adagrasib dose is 600 mg given orally twice daily until disease progression or unacceptable toxicity, according to the prescribing information. 

Adverse reactions occurring in at least 20% of treated patients included rash, nausea, diarrhea, vomiting, fatigue, musculoskeletal pain, hepatotoxicity, headache, dry skin, abdominal pain, decreased appetite, edema, anemia, cough, dizziness, constipation, and peripheral neuropathy.

“Patients with KRAS G12C–mutated colorectal cancer have historically faced poor prognoses and remain in need of additional treatment options,” Scott Kopetz, MD, PhD, of the University of Texas MD Anderson Cancer Center, Houston, stated earlier this year in a press release announcing the FDA’s decision to accept the drug application for priority review.

“Although KRAS had previously been considered ‘undruggable,’ these data from KRYSTAL-1 reinforce the potential benefit of adagrasib for these specific patients,” Dr. Kopetz said in the statement from Bristol Myers Squibb, which acquired Mirati Therapeutics in 2023.

A version of this article first appeared on Medscape.com.

The US Food and Drug Administration (FDA) has granted accelerated approval to adagrasib (Krazati, Mirati Therapeutics) with cetuximab for certain patients with KRAS G12C–mutated colorectal cancer (CRC).

More specifically, the highly selective and potent small-molecule KRAS G12C inhibitor is now indicated for patients with locally advanced or metastatic KRAS G12C–mutated CRC — as determined by an FDA-approved test — who previously received fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy and, if eligible, a vascular endothelial growth factor inhibitor, according to an FDA press release.

The agent is the first KRAS inhibitor approved for CRC. Adagrasib was previously granted accelerated approval for KRAS G12C–mutated non–small cell lung cancer, based on findings from the KRYSTAL-12 trial.

The CRC approval was based on findings from the KRYSTAL-1 multicenter, single-arm expansion cohort trial, which reported an overall response rate of 34% among 94 enrolled patients. 

All responses were partial responses, and the median duration of response was 5.8 months, with 31% of responding patients experiencing a duration of response of at least 6 months.

Patients received 600 mg of adagrasib twice daily plus cetuximab administered in either a biweekly 500 mg/m² dose or an initial dose of 400 mg/m² followed by weekly doses of 250 mg/m². Those who discontinued adagrasib also had to discontinue cetuximab, but adagrasib could be continued if cetuximab was discontinued.

The recommended adagrasib dose is 600 mg given orally twice daily until disease progression or unacceptable toxicity, according to the prescribing information. 

Adverse reactions occurring in at least 20% of treated patients included rash, nausea, diarrhea, vomiting, fatigue, musculoskeletal pain, hepatotoxicity, headache, dry skin, abdominal pain, decreased appetite, edema, anemia, cough, dizziness, constipation, and peripheral neuropathy.

“Patients with KRAS G12C–mutated colorectal cancer have historically faced poor prognoses and remain in need of additional treatment options,” Scott Kopetz, MD, PhD, of the University of Texas MD Anderson Cancer Center, Houston, stated earlier this year in a press release announcing the FDA’s decision to accept the drug application for priority review.

“Although KRAS had previously been considered ‘undruggable,’ these data from KRYSTAL-1 reinforce the potential benefit of adagrasib for these specific patients,” Dr. Kopetz said in the statement from Bristol Myers Squibb, which acquired Mirati Therapeutics in 2023.

A version of this article first appeared on Medscape.com.

The US Food and Drug Administration (FDA) has granted accelerated approval to adagrasib (Krazati, Mirati Therapeutics) with cetuximab for certain patients with KRAS G12C–mutated colorectal cancer (CRC).

More specifically, the highly selective and potent small-molecule KRAS G12C inhibitor is now indicated for patients with locally advanced or metastatic KRAS G12C–mutated CRC — as determined by an FDA-approved test — who previously received fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy and, if eligible, a vascular endothelial growth factor inhibitor, according to an FDA press release.

The agent is the first KRAS inhibitor approved for CRC. Adagrasib was previously granted accelerated approval for KRAS G12C–mutated non–small cell lung cancer, based on findings from the KRYSTAL-12 trial.

The CRC approval was based on findings from the KRYSTAL-1 multicenter, single-arm expansion cohort trial, which reported an overall response rate of 34% among 94 enrolled patients. 

All responses were partial responses, and the median duration of response was 5.8 months, with 31% of responding patients experiencing a duration of response of at least 6 months.

Patients received 600 mg of adagrasib twice daily plus cetuximab administered in either a biweekly 500 mg/m² dose or an initial dose of 400 mg/m² followed by weekly doses of 250 mg/m². Those who discontinued adagrasib also had to discontinue cetuximab, but adagrasib could be continued if cetuximab was discontinued.

The recommended adagrasib dose is 600 mg given orally twice daily until disease progression or unacceptable toxicity, according to the prescribing information. 

Adverse reactions occurring in at least 20% of treated patients included rash, nausea, diarrhea, vomiting, fatigue, musculoskeletal pain, hepatotoxicity, headache, dry skin, abdominal pain, decreased appetite, edema, anemia, cough, dizziness, constipation, and peripheral neuropathy.

“Patients with KRAS G12C–mutated colorectal cancer have historically faced poor prognoses and remain in need of additional treatment options,” Scott Kopetz, MD, PhD, of the University of Texas MD Anderson Cancer Center, Houston, stated earlier this year in a press release announcing the FDA’s decision to accept the drug application for priority review.

“Although KRAS had previously been considered ‘undruggable,’ these data from KRYSTAL-1 reinforce the potential benefit of adagrasib for these specific patients,” Dr. Kopetz said in the statement from Bristol Myers Squibb, which acquired Mirati Therapeutics in 2023.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has approved a topical anticholinergic, sofpironium topical gel, 12.45%, for the treatment of primary axillary hyperhidrosis in adults and children aged ≥ 9 years.

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According to a press release from Botanix Pharmaceuticals, which developed the product and will market it under the brand name Sofdra, approval was based on results from two phase 3 studies that enrolled 710 patients with primary axillary hyperhidrosis. In the trials, patients treated with sofpironium topical gel, 12.45%, experienced “clinically and statistically meaningful changes” from baseline in the Gravimetric Sweat Production and the Hyperhidrosis Disease Severity Measure–Axillary seven-item score, according to the company.

Botanix plans to enable qualified patients to gain early access to the product in the third quarter of 2024, with commercial sales expected in the fourth quarter of 2024.
 

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has approved a topical anticholinergic, sofpironium topical gel, 12.45%, for the treatment of primary axillary hyperhidrosis in adults and children aged ≥ 9 years.

[embed:render:related:node:263559]

According to a press release from Botanix Pharmaceuticals, which developed the product and will market it under the brand name Sofdra, approval was based on results from two phase 3 studies that enrolled 710 patients with primary axillary hyperhidrosis. In the trials, patients treated with sofpironium topical gel, 12.45%, experienced “clinically and statistically meaningful changes” from baseline in the Gravimetric Sweat Production and the Hyperhidrosis Disease Severity Measure–Axillary seven-item score, according to the company.

Botanix plans to enable qualified patients to gain early access to the product in the third quarter of 2024, with commercial sales expected in the fourth quarter of 2024.
 

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has approved a topical anticholinergic, sofpironium topical gel, 12.45%, for the treatment of primary axillary hyperhidrosis in adults and children aged ≥ 9 years.

[embed:render:related:node:263559]

According to a press release from Botanix Pharmaceuticals, which developed the product and will market it under the brand name Sofdra, approval was based on results from two phase 3 studies that enrolled 710 patients with primary axillary hyperhidrosis. In the trials, patients treated with sofpironium topical gel, 12.45%, experienced “clinically and statistically meaningful changes” from baseline in the Gravimetric Sweat Production and the Hyperhidrosis Disease Severity Measure–Axillary seven-item score, according to the company.

Botanix plans to enable qualified patients to gain early access to the product in the third quarter of 2024, with commercial sales expected in the fourth quarter of 2024.
 

A version of this article first appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved Skyrizi (risankizumab-rzaa; AbbVie) for the treatment of moderately to severely active ulcerative colitis in adults. This approval makes it the first specific anti-interleukin 23 monoclonal antibody indicated for both ulcerative colitis and moderate to severe Crohn’s disease. 

The drug is also approved in the United States for the treatment of adults with active psoriatic arthritis and moderate to severe plaque psoriasis.

The safety and efficacy of Skyrizi for ulcerative colitis is supported by data from two phase 3 clinical trials: a 12-week induction study (INSPIRE) and a 52-week maintenance study (COMMAND). 

The data showed that clinical remission, the primary endpoint in both the induction and maintenance studies, was achieved along with endoscopic improvement, which was a key secondary endpoint.

“When treating patients with ulcerative colitis, it’s important to prioritize both early and sustained clinical remission as well as endoscopic improvement,” Edward V. Loftus Jr., MD, AGAF, gastroenterologist at Mayo Clinic in Rochester, Minnesota, said in a news release. “This approval for Skyrizi is an important step toward addressing these treatment goals.”

For the treatment of ulcerative colitis, dosing includes a 12-week induction period with three 1200-mg doses delivered every 4 weeks followed by maintenance therapy of either 180 mg or 360 mg delivered every 8 weeks.

After the induction period, Skyrizi treatment can be maintained at home using an on-body injector (OBI). “The OBI is a hands-free device designed with patients in mind that adheres to the body and takes about 5 minutes to deliver the medication following preparation steps,” according to the news release. 

Full prescribing information is available online. 

A version of this article appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved Skyrizi (risankizumab-rzaa; AbbVie) for the treatment of moderately to severely active ulcerative colitis in adults. This approval makes it the first specific anti-interleukin 23 monoclonal antibody indicated for both ulcerative colitis and moderate to severe Crohn’s disease. 

The drug is also approved in the United States for the treatment of adults with active psoriatic arthritis and moderate to severe plaque psoriasis.

The safety and efficacy of Skyrizi for ulcerative colitis is supported by data from two phase 3 clinical trials: a 12-week induction study (INSPIRE) and a 52-week maintenance study (COMMAND). 

The data showed that clinical remission, the primary endpoint in both the induction and maintenance studies, was achieved along with endoscopic improvement, which was a key secondary endpoint.

“When treating patients with ulcerative colitis, it’s important to prioritize both early and sustained clinical remission as well as endoscopic improvement,” Edward V. Loftus Jr., MD, AGAF, gastroenterologist at Mayo Clinic in Rochester, Minnesota, said in a news release. “This approval for Skyrizi is an important step toward addressing these treatment goals.”

For the treatment of ulcerative colitis, dosing includes a 12-week induction period with three 1200-mg doses delivered every 4 weeks followed by maintenance therapy of either 180 mg or 360 mg delivered every 8 weeks.

After the induction period, Skyrizi treatment can be maintained at home using an on-body injector (OBI). “The OBI is a hands-free device designed with patients in mind that adheres to the body and takes about 5 minutes to deliver the medication following preparation steps,” according to the news release. 

Full prescribing information is available online. 

A version of this article appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved Skyrizi (risankizumab-rzaa; AbbVie) for the treatment of moderately to severely active ulcerative colitis in adults. This approval makes it the first specific anti-interleukin 23 monoclonal antibody indicated for both ulcerative colitis and moderate to severe Crohn’s disease. 

The drug is also approved in the United States for the treatment of adults with active psoriatic arthritis and moderate to severe plaque psoriasis.

The safety and efficacy of Skyrizi for ulcerative colitis is supported by data from two phase 3 clinical trials: a 12-week induction study (INSPIRE) and a 52-week maintenance study (COMMAND). 

The data showed that clinical remission, the primary endpoint in both the induction and maintenance studies, was achieved along with endoscopic improvement, which was a key secondary endpoint.

“When treating patients with ulcerative colitis, it’s important to prioritize both early and sustained clinical remission as well as endoscopic improvement,” Edward V. Loftus Jr., MD, AGAF, gastroenterologist at Mayo Clinic in Rochester, Minnesota, said in a news release. “This approval for Skyrizi is an important step toward addressing these treatment goals.”

For the treatment of ulcerative colitis, dosing includes a 12-week induction period with three 1200-mg doses delivered every 4 weeks followed by maintenance therapy of either 180 mg or 360 mg delivered every 8 weeks.

After the induction period, Skyrizi treatment can be maintained at home using an on-body injector (OBI). “The OBI is a hands-free device designed with patients in mind that adheres to the body and takes about 5 minutes to deliver the medication following preparation steps,” according to the news release. 

Full prescribing information is available online. 

A version of this article 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.

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 US Food and Drug Administration has expanded the indication for durvalumab (Imfinzi, AstraZeneca) to include mismatch repair deficient (dMMR) newly diagnosed advanced or recurrent endometrial cancer in combination with carboplatin and paclitaxel followed by single-agent use for maintenance.

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.

The US Food and Drug Administration has expanded the indication for durvalumab (Imfinzi, AstraZeneca) to include mismatch repair deficient (dMMR) newly diagnosed advanced or recurrent endometrial cancer in combination with carboplatin and paclitaxel followed by single-agent use for maintenance.

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.

The US Food and Drug Administration has expanded the indication for durvalumab (Imfinzi, AstraZeneca) to include mismatch repair deficient (dMMR) newly diagnosed advanced or recurrent endometrial cancer in combination with carboplatin and paclitaxel followed by single-agent use for maintenance.

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.

The US Food and Drug Administration (FDA) has granted accelerated approval for Iqirvo (elafibranor; Ipsen) for treatment of primary biliary cholangitis (PBC) 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.

The US Food and Drug Administration (FDA) has granted accelerated approval for Iqirvo (elafibranor; Ipsen) for treatment of primary biliary cholangitis (PBC) 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.

The US Food and Drug Administration (FDA) has granted accelerated approval for Iqirvo (elafibranor; Ipsen) for treatment of primary biliary cholangitis (PBC) 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.

The US Food and Drug Administration has granted accelerated approval to repotrectinib (Augtyro, Bristol Myers Squibb) for all locally advanced, unresectable, or metastatic solid tumors with an NTRK gene fusion that have progressed after initial treatment or that have no satisfactory alternative therapies.

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 US Food and Drug Administration has granted accelerated approval to repotrectinib (Augtyro, Bristol Myers Squibb) for all locally advanced, unresectable, or metastatic solid tumors with an NTRK gene fusion that have progressed after initial treatment or that have no satisfactory alternative therapies.

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 US Food and Drug Administration has granted accelerated approval to repotrectinib (Augtyro, Bristol Myers Squibb) for all locally advanced, unresectable, or metastatic solid tumors with an NTRK gene fusion that have progressed after initial treatment or that have no satisfactory alternative therapies.

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 US Food and Drug Administration has granted accelerated approval to tarlatamab-dlle (Imdelltra) for extensive-stage small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy.

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: aotto@mdedge.com

A version of this article appeared on Medscape.com.

The US Food and Drug Administration has granted accelerated approval to tarlatamab-dlle (Imdelltra) for extensive-stage small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy.

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: aotto@mdedge.com

A version of this article appeared on Medscape.com.

The US Food and Drug Administration has granted accelerated approval to tarlatamab-dlle (Imdelltra) for extensive-stage small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy.

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: aotto@mdedge.com

A version of this article appeared on Medscape.com.

The US Food and Drug Administration (FDA) has granted accelerated approval to lisocabtagene maraleucel (Breyanzi, Juno Therapeutics /Bristol Myers Squibb) for adults with relapsed or refractory follicular lymphoma who have received two or more prior lines of systemic therapy.

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 US Food and Drug Administration (FDA) has granted accelerated approval to lisocabtagene maraleucel (Breyanzi, Juno Therapeutics /Bristol Myers Squibb) for adults with relapsed or refractory follicular lymphoma who have received two or more prior lines of systemic therapy.

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 US Food and Drug Administration (FDA) has granted accelerated approval to lisocabtagene maraleucel (Breyanzi, Juno Therapeutics /Bristol Myers Squibb) for adults with relapsed or refractory follicular lymphoma who have received two or more prior lines of systemic therapy.

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.