FDA NEWS: November 2020

TOP - November 2020 Vol 13, No 6 - FDA Updates

This section provides a brief overview of new cancer drugs approved by the FDA between July 31, 2020, and September 4, 2020.

Gavreto FDA Approved for Metastatic NSCLC with RET Fusions

On September 4, 2020, the FDA accelerated the approval of pralsetinib (Gavreto; Blueprint Medicines/Genentech), an oral RET inhibitor, for the treatment of adults with metastatic non–small-cell lung cancer (NSCLC) and RET-activating fusions, as detected by an FDA-approved test.

The FDA granted pralsetinib a breakthrough therapy designation for the treatment of NSCLC and RET fusion that has progressed after platinum-based chemotherapy, as well as for patients with medullary thyroid cancer and RET mutation that requires systemic treatment and for which there are no acceptable alternative treatments.

RET-activating fusions and mutations are key drivers in many cancer types, including NSCLC and medullary thyroid cancer. Currently, there are limited targeted treatments for patients with these types of genetic alterations. Approximately 1% to 2% of people with NSCLC have RET fusions. Biomarker testing for these fusions is the most effective way to identify people who are eligible for treatment with pralsetinib.

“The FDA approval of Gavreto for RET fusion-positive non–small-cell lung cancer is an important step towards our goal of providing an effective treatment option for every person diagnosed with lung cancer, no matter how rare or hard-to-treat their type of disease,” said Levi Garraway, MD, PhD, Chief Medical Officer and Head of Global Product Development at Genentech. “We remain committed to finding personalized treatment options for people with cancer based on specific genomic or molecular alterations, and we look forward to partnering with Blueprint Medicines to further explore the potential of Gavreto across multiple RET-altered tumor types.”

The approval of pralsetinib for this indication was based on the results of the phase 1/2 ARROW clinical trial, a first-in-human study that is evaluating the safety and efficacy of pralsetinib in patients with NSCLC or thyroid cancer that is associated with RET fusions or mutations. Patients receive 400 mg of pralsetinib orally once daily.

Among the 87 patients with NSCLC who have previously received platinum-based chemotherapy, the overall response rate (ORR) with pralsetinib was 57% (95% confidence interval [CI], 46%-68%), including a 5.7% complete response rate. The median duration of response has not been reached (95% CI, 15.2 months-not reached).

In the 27 patients with treatment-naïve NSCLC, the ORR was 70% (95% CI, 50%-86%), including an 11% complete response rate.

The most common (≥25%) adverse reactions were fatigue, constipation, musculoskeletal pain, and hypertension.

Blueprint Medicines will offer the YourBlueprint program as a patient assistance program for eligible patients who are prescribed pralsetinib and need financial assistance.


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Blenrep Receives FDA Approval for Relapsed or Refractory Multiple Myeloma

On August 5, 2020, the FDA accelerated the approval of belantamab mafodotin-blmf (Blenrep; GlaxoSmithKline), a B-cell maturation antigen (BCMA)-directed antibody and microtubule inhibitor conjugate, for the treatment of adults with relapsed or refractory multiple myeloma who have received ≥4 previous therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent.

The FDA granted belantamab mafodotin an orphan drug designation and a breakthrough therapy designation for this indication.

The approval of belantamab mafodotin-blmf was based on the results of the DREAMM-2 study, an open-label, multicenter clinical trial. Patients received belantamab mafodotin-blmf 2.5 mg/kg or 3.4 mg/kg intravenously once every 3 weeks until disease progression or unacceptable toxicity.

The efficacy end point included ORR and response duration, as evaluated by an Independent Review Committee using the International Myeloma Working Group uniform response criteria. The ORR was 31% (97.5% CI, 21%-43%). Overall, 73% of those who responded to therapy with the 2.5-mg/kg dose of belantamab mafodotin had a response lasting ≥6 months.

The most common (≥20%) adverse reactions to belantamab mafodotin-blmf therapy included keratopathy, decreased visual acuity, nausea, blurred vision, pyrexia, infusion-related reactions, and fatigue.

Because belantamab mafodotin is associated with a risk for serious ocular adverse events, including changes in the corneal epithelium resulting in severe vision loss and corneal ulcer, in addition to blurred vision and dry eyes, the drug is only available through a Risk Evaluation and Mitigation Strategy (REMS), the BLENREP REMS.

The recommended dose of belantamab mafodotin-blmf is 2.5 mg/kg as an intravenous infusion over approximately 30 minutes once every 3 weeks.


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FDA Approves Monjuvi plus Revlimid for Relapsed or Refractory Diffuse Large B-Cell Lymphoma

On July 31, 2020, the FDA accelerated the approval of tafasitamab-cxix (Monjuvi; Incyte/MorphoSys US), CD19-directed cytolytic antibody, in combination with lenalidomide (Revlimid) for the treatment of adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low-grade lymphoma, who are not eligible for autologous stem-cell transplant.

The FDA previously granted a breakthrough therapy designation for the combination of tafasitamab and lenalidomide for this indication.

“The FDA approval of Monjuvi brings a new treatment option to patients in dire need across the United States,” said Professor Gilles Salles, MD, Chair of the Clinical Hematology Department, University of Lyon, France, and lead investigator of the L-MIND study. “Today’s FDA decision offers new hope for patients with this aggressive form of DLBCL who progressed during or after first-line therapy.”

The approval of tafasitamab was based on the results of the phase 2 L-MIND study, an open-label, multicenter, single-arm clinical trial. The study included patients with relapsed or refractory DLBCL who received treatment with tafasitamab in combination with lenalidomide.

The primary end point was ORR. The ORR was 55%, including a 37% rate of complete response and an 18% partial response rate. The median duration of response was 21.7 months.

The most common (≥20%) adverse reactions were neutropenia, fatigue, anemia, diarrhea, thrombocytopenia, cough, pyrexia, peripheral edema, respiratory tract infection, and decreased appetite.

For eligible patients who require financial assistance, Incyte and MorphoSys US offer a patient support program, My Mission Support.


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Last modified: November 10, 2020