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FDA Approvals in GVHD, CRC, and AML, a Priority Review in MCL, and More

Gina Columbus
Published Online: Wednesday, Aug 09, 2017



Today-

FDA approvals in graft-versus-host disease, colorectal cancer, and acute myeloid leukemia, a priority review in mantle cell lymphoma, breakthrough designations in AML and non–small cell lung cancer, and a European approval in breast cancer.

Welcome to OncLive News Network! I’m Gina Columbus.

The FDA has approved ibrutinib for the treatment of adult patients with chronic graft-versus-host disease, following the failure of 1 or more lines of systemic therapy. This decision marks the first FDA-approved therapy for cGVHD.

The approval is based on data from the single-arm phase Ib/II PCYC-1129 trial, in which the BTK inhibitor induced an overall response rate of 67% and showed clinically meaningful and durable responses in patients who failed at least 1 prior treatment for cGVHD.

Twenty-one percent of responders had a complete response and 45% had a partial response. Additionally, most responders were able to reduce steroid doses to an acceptable minimal level.

The FDA granted a breakthrough therapy designation to ibrutinib for this indication in June 2016.

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The FDA granted an accelerated approval to nivolumab for the treatment of adult and pediatric patients with microsatellite instability-high or mismatch repair deficient metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan.

The decision is based on findings from the phase II CheckMate-142 trial, in which the overall response rate was 28% in patients with mCRC who received prior therapy, including 1 complete response and 14 partial responses. In the open-label, international study, patients were randomized to receive either single-agent nivolumab or nivolumab combined with ipilimumab, and the FDA reviewed the nivolumab-only cohort.

The FDA-recommended dose for the PD-1 inhibitor in this setting is 240 mg IV every 2 weeks until disease progression or unacceptable toxicity. The accelerated approval of nivolumab for this indication is contingent upon the outcomes of confirmatory trials.

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In acute myeloid leukemia, the FDA approved enasidenib as a treatment for patients with relapsed or refractory IDH2-mutated disease.

The approval was based on findings from the phase I/II AG221-C-001 trial, in which enasidenib demonstrated a complete remission rate of 19.3%. The median duration of CR was 8.2 months, and 4% of patients had a CR with partial hematologic recovery, which lasted for 9.6 months, according to the FDA.

Thirty-four percent of 157 transplant-dependent patients could stop receiving blood or platelet transfusions following treatment with enasidenib.

A companion diagnostic, the RealTime IDH2 Assay, was also approved for the detection of the IDH2 mutation.

******************************************

The FDA has granted a priority review to a new drug application for acalabrutinib for patients with previously treated mantle cell lymphoma.

The announcement came just 1 day after AstraZeneca, the manufacturer of the BTK inhibitor, reported that acalabrutinib had received an FDA breakthrough therapy designation in this setting. The NDA is supported, in part, with phase II results from the ACE-LY-004 trial, which evaluated the safety and efficacy of acalabrutinib in patients with relapsed/refractory MCL.

The company has not made any data from that trial available, but a spokesperson wrote in an email to OncLive that the data is slated to be presented at a medical meeting later this year.

Under the Prescription Drug User Fee Act, the FDA is scheduled to issue its final approval decision for acalabrutinib in this setting by early next year.

*****************************************

Venetoclax has received a breakthrough therapy designation by the FDA for use in combination with low-dose cytarabine in treatment-naïve elderly patients with acute myeloid leukemia who are ineligible for intensive chemotherapy.

The designation, which is the fourth for venetoclax, will expedite the development and review of the combination in this AML subgroup. The status was granted based on findings from the phase I/II M14-387 study that were presented at the 2017 European Hematology Association Annual Congress.

In the study, the overall response rate was 61%, which included a complete remission rate of 21%, a CR with incomplete blood count recovery rate of 33%, and a partial remission rate of 7%.

Activity with the venetoclax combination was observed across subgroups. Additionally, the researchers reported that survival was longer among patients who achieved a response.

*******************************

The FDA has granted durvalumab a breakthrough therapy designation to treat patients with locally advanced, unresectable non-small cell lung cancer whose disease has not progressed following platinum-based chemoradiation.

The designation is based on interim results from the double-blind, phase III PACIFIC trial, in which the PD-L1 inhibitor significantly improved progression-free survival versus placebo. AstraZeneca, the manufacturer of durvalumab, has not released any details from the study, but said data have been submitted for presentation at an upcoming medical meeting.

PACIFIC was conducted at 235 centers in 26 countries and included an “all-comer” patient population who had locally-advanced, unresectable stage III NSCLC with no disease progression after concurrent therapy with platinum-based chemotherapy and radiation.

Patients were randomized in a 2:1 ratio to durvalumab or placebo. PFS was the primary endpoint, along with overall survival.

*******************************

The European Commission has approved the use of fulvestrant to treat estrogen receptor-positive, locally advanced or metastatic breast cancer in postmenopausal women who are not previously treated with endocrine therapy.

The EC based its decision on results from the phase III FALCON trial, which showed that fulvestrant extended median progression-free survival by 2.8 months compared with anastrozole.

Fulvestrant has been approved in the United States since 2002 for the treatment of ER-positive, locally advanced or metastatic breast cancer in postmenopausal women not previously treated with endocrine therapy, or with disease relapse on or after adjuvant anti-estrogen therapy, or disease progression on anti-estrogen therapy.

In February 2016, the FDA approved fulvestrant in combination with palbociclib for women with HR-positive, HER2-negative advanced or metastatic breast cancer, whose cancer has progressed after endocrine therapy.

*******************************

That’s all for today.

Thank you for watching OncLive News Network! I’m Gina Columbus.
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Today-

FDA approvals in graft-versus-host disease, colorectal cancer, and acute myeloid leukemia, a priority review in mantle cell lymphoma, breakthrough designations in AML and non–small cell lung cancer, and a European approval in breast cancer.

Welcome to OncLive News Network! I’m Gina Columbus.

The FDA has approved ibrutinib for the treatment of adult patients with chronic graft-versus-host disease, following the failure of 1 or more lines of systemic therapy. This decision marks the first FDA-approved therapy for cGVHD.

The approval is based on data from the single-arm phase Ib/II PCYC-1129 trial, in which the BTK inhibitor induced an overall response rate of 67% and showed clinically meaningful and durable responses in patients who failed at least 1 prior treatment for cGVHD.

Twenty-one percent of responders had a complete response and 45% had a partial response. Additionally, most responders were able to reduce steroid doses to an acceptable minimal level.

The FDA granted a breakthrough therapy designation to ibrutinib for this indication in June 2016.

*****************************************

The FDA granted an accelerated approval to nivolumab for the treatment of adult and pediatric patients with microsatellite instability-high or mismatch repair deficient metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan.

The decision is based on findings from the phase II CheckMate-142 trial, in which the overall response rate was 28% in patients with mCRC who received prior therapy, including 1 complete response and 14 partial responses. In the open-label, international study, patients were randomized to receive either single-agent nivolumab or nivolumab combined with ipilimumab, and the FDA reviewed the nivolumab-only cohort.

The FDA-recommended dose for the PD-1 inhibitor in this setting is 240 mg IV every 2 weeks until disease progression or unacceptable toxicity. The accelerated approval of nivolumab for this indication is contingent upon the outcomes of confirmatory trials.

********************************

In acute myeloid leukemia, the FDA approved enasidenib as a treatment for patients with relapsed or refractory IDH2-mutated disease.

The approval was based on findings from the phase I/II AG221-C-001 trial, in which enasidenib demonstrated a complete remission rate of 19.3%. The median duration of CR was 8.2 months, and 4% of patients had a CR with partial hematologic recovery, which lasted for 9.6 months, according to the FDA.

Thirty-four percent of 157 transplant-dependent patients could stop receiving blood or platelet transfusions following treatment with enasidenib.

A companion diagnostic, the RealTime IDH2 Assay, was also approved for the detection of the IDH2 mutation.

******************************************

The FDA has granted a priority review to a new drug application for acalabrutinib for patients with previously treated mantle cell lymphoma.

The announcement came just 1 day after AstraZeneca, the manufacturer of the BTK inhibitor, reported that acalabrutinib had received an FDA breakthrough therapy designation in this setting. The NDA is supported, in part, with phase II results from the ACE-LY-004 trial, which evaluated the safety and efficacy of acalabrutinib in patients with relapsed/refractory MCL.

The company has not made any data from that trial available, but a spokesperson wrote in an email to OncLive that the data is slated to be presented at a medical meeting later this year.

Under the Prescription Drug User Fee Act, the FDA is scheduled to issue its final approval decision for acalabrutinib in this setting by early next year.

*****************************************

Venetoclax has received a breakthrough therapy designation by the FDA for use in combination with low-dose cytarabine in treatment-naïve elderly patients with acute myeloid leukemia who are ineligible for intensive chemotherapy.

The designation, which is the fourth for venetoclax, will expedite the development and review of the combination in this AML subgroup. The status was granted based on findings from the phase I/II M14-387 study that were presented at the 2017 European Hematology Association Annual Congress.

In the study, the overall response rate was 61%, which included a complete remission rate of 21%, a CR with incomplete blood count recovery rate of 33%, and a partial remission rate of 7%.

Activity with the venetoclax combination was observed across subgroups. Additionally, the researchers reported that survival was longer among patients who achieved a response.

*******************************

The FDA has granted durvalumab a breakthrough therapy designation to treat patients with locally advanced, unresectable non-small cell lung cancer whose disease has not progressed following platinum-based chemoradiation.

The designation is based on interim results from the double-blind, phase III PACIFIC trial, in which the PD-L1 inhibitor significantly improved progression-free survival versus placebo. AstraZeneca, the manufacturer of durvalumab, has not released any details from the study, but said data have been submitted for presentation at an upcoming medical meeting.

PACIFIC was conducted at 235 centers in 26 countries and included an “all-comer” patient population who had locally-advanced, unresectable stage III NSCLC with no disease progression after concurrent therapy with platinum-based chemotherapy and radiation.

Patients were randomized in a 2:1 ratio to durvalumab or placebo. PFS was the primary endpoint, along with overall survival.

*******************************

The European Commission has approved the use of fulvestrant to treat estrogen receptor-positive, locally advanced or metastatic breast cancer in postmenopausal women who are not previously treated with endocrine therapy.

The EC based its decision on results from the phase III FALCON trial, which showed that fulvestrant extended median progression-free survival by 2.8 months compared with anastrozole.

Fulvestrant has been approved in the United States since 2002 for the treatment of ER-positive, locally advanced or metastatic breast cancer in postmenopausal women not previously treated with endocrine therapy, or with disease relapse on or after adjuvant anti-estrogen therapy, or disease progression on anti-estrogen therapy.

In February 2016, the FDA approved fulvestrant in combination with palbociclib for women with HR-positive, HER2-negative advanced or metastatic breast cancer, whose cancer has progressed after endocrine therapy.

*******************************

That’s all for today.

Thank you for watching OncLive News Network! I’m Gina Columbus.
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