FDA has approved pralsetinib for the treatment of patients with advanced or metastatic RET-mutant an

On December 1, 2020, South San Francisco, California – Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration (FDA) has approved Gavreto™ (pralsetinib) for the treatment of adult and pediatric patients aged 12 years and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and are radioiodine-refractory (if radioiodine therapy is appropriate). These indications were approved under the FDA’s Accelerated Approval Program, based on data from the Phase I/II ARROW study. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

“We are honored to collaborate with Blueprint Medicines to bring this important new option to patients with specific RET-altered thyroid cancers,” said Dr. Levi Garraway, Chief Medical Officer and Head of Global Product Development. “Gavreto is now approved for multiple RET-altered tumor types, underscoring our commitment to advancing personalized healthcare through treatments that target the underlying biology of each patient’s cancer.”

Approximately 10-20% of patients with papillary thyroid cancer (the most common type of thyroid cancer) have RET fusion-positive tumors, while around 90% of patients with advanced MTC (a rare form of thyroid cancer) harbor RET mutations. Biomarker testing for RET fusions and mutations helps identify patients who are eligible for treatment with Gavreto.

This approval is based on results from the Phase I/II ARROW study, which demonstrated that Gavreto exhibits durable clinical activity in patients regardless of prior treatment history and RET alteration genotype. Among 55 patients with RET-mutant metastatic MTC who had previously received cabozantinib and/or vandetanib, the overall response rate (ORR) with Gavreto was 60% (95% CI: 46%, 73%), and the median duration of response (DoR) was not reached (95% CI: 15.1 months, not estimable). In 29 patients with RET-mutant advanced MTC who had not received prior cabozantinib or vandetanib, the ORR was 66% (95% CI: 46%, 82%), with median DoR not reached (95% CI: not estimable, not estimable). In 9 patients with RET fusion-positive metastatic thyroid cancer, the ORR with Gavreto was 89% (95% CI: 52%, 100%), and median DoR was not reached (95% CI: not estimable, not estimable). In the ARROW trial, across all patients with RET-altered tumor types, the most common adverse reactions (≥25%) included constipation, hypertension (elevated blood pressure), fatigue, musculoskeletal pain, and diarrhea.