The Food and Drug Administration (FDA) has granted accelerated approval to Gavreto™ (pralsetinib; Blueprint Medicines and Genentech) for the treatment of adults with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test.
Gavreto is a once-daily oral tyrosine kinase inhibitor designed to selectively and potently inhibit RET alterations. The approval was based on efficacy and safety data from the multicenter, open-label, multi-cohort phase 1/2 ARROW study in patients with metastatic RET fusion-positive NSCLC who were previously treated with platinum-based chemotherapy or treatment-naïve. The major efficacy outcome measures were overall response rate (ORR) and duration of response (DoR).
Among 87 patients who were previously treated with platinum-based chemotherapy, Gavreto demonstrated an ORR of 57% (95% CI, 46-68); 5.7% of patients had a complete response, and 52% had a partial response. The median DoR was not reached (95% CI, 15.2 months-not estimable), but 80% of responders had a DoR of ≥6 months.
Among 27 treatment-naïve patients, Gavreto demonstrated an ORR of 70% (95% CI, 50-86); 11% of patients had a complete response, and 59% had a partial response. The median DoR was not reached (95% CI, 6.3 months-not estimable), but 58% of responders had a DoR of ≥6 months.
As for safety, the most common adverse reactions (≥25%) were fatigue, constipation, musculoskeletal pain and increased blood pressure. In addition, Gavreto includes warnings and precautions related to interstitial lung disease/pneumonitis, hypertension, hepatotoxicity, hemorrhagic events, risk of impaired wound healing and risk of embryo-fetal toxicity.
Continued approval of Gavreto for the treatment of metastatic RET fusion-positive NSCLC may be contingent upon verification and description of clinical benefit in confirmatory trials.
“We applaud therapeutic advancements like Gavreto that allow lung cancer treatment to be personalized based on the molecular drivers in a person’s tumor,” said Andrea Ferris, President and CEO of LUNGevity. “There are now a number of tumor-specific gene alterations that can be targeted with FDA-approved therapies, reflecting an important inflection point supporting the widespread use of comprehensive biomarker testing. At LUNGevity, we want to empower patients and their families to discuss biomarker testing with clinicians prior to initiating treatment.”
Gavreto will be available within one week as 100mg capsules of pralsetinib in 60 and 90-count bottles. Blueprint Medicines will also be offering patient assistance programs for eligible patients.
For more information visit blueprintmedicines.com.
- Genentech announces FDA approval of Gavreto (pralsetinib) for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer. https://www.businesswire.com/news/home/20200904005515/en/Genentech-Announces-FDA-Approval-Gavreto-pralsetinib-Treatment. Accessed September 8, 2020.
- Blueprint Medicines announces FDA approval of Gavreto™ (pralsetinib) for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer. http://ir.blueprintmedicines.com/news-releases/news-release-details/blueprint-medicines-announces-fda-approval-gavretotm-pralsetinib. Accessed September 8, 2020.
- Gavreto [package insert]. Cambridge, MA: Blueprint Medicines Corporation; 2020.