Verastem Oncology has gained its first FDA approval, winning an accelerated nod for the combination treatment of avutometinib and defactinib for a rare form of ovarian cancer.

On May 8, 2025, the U.S. Food and Drug Administration (FDA) granted accelerated approval to Verastem Oncology's Avmapki™ Fakzynja™ Co-Pack, marking the first-ever FDA-approved treatment specifically for adults with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy . 

U.S. Food and Drug Administration About the Treatment The Avmapki Fakzynja Co-Pack combines two oral drugs: avutometinib, a MEK inhibitor, and defactinib, a FAK inhibitor. This dual approach targets the MAPK signaling pathway, which is often dysregulated in LGSOC due to KRAS mutations . The FDA's decision was based on data from the RAMP-201 Phase 2 trial involving 57 patients with KRAS-mutated recurrent LGSOC. Participants received avutometinib 3.2 mg orally twice weekly and defactinib 200 mg orally twice daily for the first three weeks of each four-week cycle. The trial reported an overall response rate of 44%, with responses lasting between 3.3 to 31.1 months .  Availability LGSOC is a rare ovarian cancer subtype, affecting approximately 6,000 to 8,000 women in the U.S. and about 80,000 globally. It predominantly affects younger women and has limited response to conventional chemotherapy . Verastem plans to make the Avmapki Fakzynja Co-Pack available within a week of approval, with a 28-day treatment cycle priced at $48,500 .  Common side effects observed include nausea, fatigue, rash, diarrhea, and elevated liver enzymes. Patients will require monitoring for potential liver toxicity, skin reactions, muscle issues, and vision problems during treatment . WebMD Future Directions The approval is contingent upon confirmatory results from the ongoing RAMP 301 Phase 3 trial, which compares the combination therapy to standard chemotherapy or hormonal treatments in patients with recurrent LGSOC . This milestone offers a promising new option for patients with this challenging cancer subtype.