Gavreto, RET Inhibitor, FDA Approved for Metastatic NSCLC with RET Fusions

Web Exclusives - FDA Updates

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 FDA approved pralsetinib for this indication 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 people 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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Last modified: November 16, 2020