FDA approval of Gavreto to treat RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. -Blueprint Medicines + Genentech/Roche

Blueprint Medicines Corporation announced that the FDA has approved Gavreto (pralsetinib) for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. The approval is based on data from the Phase 1/II ARROW clinical trial, which showed efficacy for Gavreto in patients with RET fusion-positive NSCLC with or without prior therapy, and regardless of RET fusion partner or central nervous system involvement. Under Blueprint Medicines' collaboration with Roche, Blueprint Medicines and Genentech, a member of the Roche Group, will co-commercialize Gavreto in the U.S. Gavreto was granted accelerated approval by the FDA, and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. In 87 patients previously treated with platinum-based chemotherapy, the overall response rate (ORR) was 57 percent (95% CI: 46%, 68%) with a 5.7 percent complete response (CR) rate, and the median duration of response (DOR) was not estimable (95% CI: 15.2 months, not estimable). In 27 treatment-naïve patients who were ineligible for platinum-based chemotherapy per the study protocol, the ORR was 70 percent (95% CI: 50%, 86%) with an 11 percent CR rate, and the median DOR was 9.0 months (95% CI: 6.3 months, not estimable). Gavreto has warnings and precautions of interstitial lung disease/pneumonitis, hypertension, hepatotoxicity, hemorrhagic events, risk of impaired wound healing and risk of embryo-fetal toxicity. Blueprint Medicines and Genentech plan to make Gavreto available in the U.S. within one week. Gavreto will be available in a 100 mg dose strength, and the recommended starting dose is 400 mg once daily.