FDA Approves Verastem Therapy for Rare Ovarian Cancer

The FDA has granted accelerated approval to Verastem Oncology's Avmapki Fakzynja Co-Pack, combining avutometinib and defactinib, as the first treatment for adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. The approval is based on the Phase 2 RAMP 201 trial, which demonstrated a 44% overall response rate and response durations ranging from 3.3 to 31.1 months. LGSOC is a rare cancer that often affects younger women and is resistant to both chemotherapy and hormone therapies. The approval was granted ahead of the anticipated regulatory review deadline, and Verastem expects to launch the therapy in the U.S. within a week, marking its transition to a commercial-stage company. The company is conducting a confirmatory Phase 3 trial and recently raised $75 million to support the launch and additional research. Verastem's stock price rose notably following the announcement.