BOSTON--Verastem Oncology, a biopharmaceutical company dedicated to developing new cancer treatments, has received FDA acceptance and Priority Review for its New Drug Application (NDA) for avutometinib combined with defactinib. This treatment targets adult patients with recurrent low-grade serous ovarian cancer (LGSOC) who have a KRAS mutation and have undergone at least one prior systemic therapy. The NDA, submitted in October 2024, has a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2025.
Key Highlights:
- Priority Review indicates the FDA's recognition of the significant unmet need for effective treatments for recurrent KRAS mutant LGSOC.
- Clinical Trial Results: The NDA submission is supported by data from the Phase 2 RAMP 201 clinical trial, which demonstrated a substantial overall response rate and durable responses in patients treated with the combination of avutometinib and defactinib.
- Current Treatment Landscape: There are currently no FDA-approved treatments specifically for LGSOC, highlighting the potential impact of this new therapy.
About the Treatment:
- Avutometinib is an oral RAF/MEK clamp that inhibits MEK1/2 kinase activities, potentially creating a more complete and durable anti-tumor response.
- Defactinib is an oral, selective inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2), which are involved in cancer cell proliferation, survival, migration, and invasion.
Future Steps: Verastem Oncology is currently enrolling patients for RAMP 301, an international Phase 3 trial, which will serve as a confirmatory study for the initial indication and has the potential to support an expanded indication regardless of KRAS mutation status.
About LGSOC: Low-Grade Serous Ovarian Cancer (LGSOC) is a rare and distinct form of ovarian cancer that is less sensitive to chemotherapy and highly recurrent. It affects approximately 6,000-8,000 women in the U.S. and 80,000 worldwide, with a median survival of approximately ten years.
This development represents a significant step forward in addressing a condition that has long been overlooked, offering hope to patients with recurrent KRAS mutant LGSOC.