Verastem Oncology’s New Drug Application (NDA) for a combination therapy of avutometinib and defactinib, designed to treat recurrent low-grade serous ovarian cancer (LGSOC) with a KRAS mutation, has been accepted for Priority Review by the FDA. The FDA has set a target action date of June 30, 2025, and if approved, this would be the first FDA-approved treatment specifically for this patient population. The application is based on positive results from the Phase 2 RAMP 201 clinical trial, which showed substantial overall response rates and durable responses in patients with recurrent KRAS mutant LGSOC.
This FDA acceptance is crucial for patients with LGSOC, a rare and often fatal ovarian cancer distinct from its high-grade counterpart. Currently, no FDA-approved treatments exist specifically for LGSOC, leaving patients with limited options. The combination of avutometinib, a RAF/MEK clamp, and defactinib, a FAK inhibitor, offers a potential breakthrough for these patients, particularly those with the KRAS mutation, by targeting specific pathways that drive cancer cell survival and tumor growth. The Priority Review designation further underscores the unmet need and the potential impact this therapy could have.
The NDA submission includes data from the Phase 2 RAMP 201 trial, highlighting the combination therapy’s efficacy and tolerability. Supportive data from the earlier Phase 1 FRAME trial is also included. A Phase 3 confirmatory trial, RAMP 301, is currently enrolling patients with recurrent LGSOC, regardless of KRAS mutation status. This trial will not only confirm the findings of the Phase 2 study but could also potentially expand the treatment’s indication to include patients without the KRAS mutation.
A potential FDA approval in June 2025 would mark a significant milestone for Verastem Oncology and, more importantly, for patients battling recurrent LGSOC. It would provide a much-needed targeted treatment option and could pave the way for further research and development in this area. The ongoing Phase 3 trial has the potential to broaden the treatment’s applicability and benefit a larger population of LGSOC patients. This progress could significantly alter the treatment landscape for this challenging cancer.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
