Blueprint Medicines recently announced that the U.S. Food and Drug Administration (FDA) has approved Gavreto (pralsetinib), a precision oncology drug, for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) who are confirmed to have RET fusion-positive tumors by an FDA-approved testing method.
On the same day, the FDA also accepted a New Drug Application (NDA) for Gavreto for the treatment of RET-mutant medullary thyroid carcinoma (MTC) and RET fusion-positive thyroid cancer, which will be reviewed through the Real-Time Oncology Review (RTOR) pilot program. The FDA has granted the NDA priority review, with a Prescription Drug User Fee Act (PDUFA) target date of February 28, 2021.
Notably, Gavreto is the only once-daily oral RET-targeted therapy that has demonstrated durable efficacy and a high complete response rate in patients with RET fusion-positive NSCLC. This approval is based on tumor response data from the Phase I/II ARROW study, which showed that Gavreto was effective in patients with RET fusion-positive NSCLC regardless of RET fusion partner or central nervous system involvement, with a subset of patients achieving complete remission.
RET biomarker detection is the only method to identify eligibility for Gavreto treatment in patients with metastatic NSCLC. RET fusions can be identified using available biomarker detection methods, including next-generation sequencing of tumor tissue or liquid biopsies. In the ARROW trial, next-generation sequencing, FISH, or other methods were used to detect RET fusions.
Based on tumor response data from the Phase I/II ARROW study, Gavreto received accelerated approval from the FDA. Continued approval for this indication will depend on the validation and description of clinical benefit in confirmatory clinical trials. In this study, patients with RET fusion-positive NSCLC received Gavreto at a dose of 400 mg once daily. Data showed:
(1) In 87 patients who had previously received platinum-based chemotherapy, the objective response rate (ORR) was 57% (95% CI: 46-68%), the complete response rate (CR) was 5.7%, and the median duration of response (DOR) had not yet been reached (95% CI: 15.2 months, not estimable).
(2) In 27 treatment-naïve patients who had not received platinum-based chemotherapy, the ORR was 70% (95% CI: 50-86%), the CR was 11%, and the median DOR was 9.0 months (95% CI: 6.3 months, not estimable).
Gavreto's drug label contains warnings and precautions regarding interstitial lung disease/pneumonia, hypertension, hepatotoxicity, bleeding events, risk of impaired wound healing, and embryo-fetal toxicity.
The above is the latest information regarding the FDA approval of Gavreto (pralsetinib), a new RET drug for non-small cell lung cancer, for once-daily oral administration. For more information about Gavreto (pralsetinib), please contact our pharmacist customer service directly.
