Defactinib FDA Approval Status
Last updated by Judith Stewart, BPharm on Jan 1, 2025.
FDA Approved: No
Generic name: defactinib
Company: Verastem Oncology
Treatment for: Ovarian Cancer
Defactinib is a selective focal adhesion kinase (FAK) inhibitor in development for use in combination with avutometinib for the treatment of adults with recurrent KRAS mutant low-grade serous ovarian cancer.
- Low-grade serous ovarian cancer (LGSOC) is a rare and distinct ovarian cancer that is persistent and ultimately fatal. LGSOC is highly recurrent and less sensitive to chemotherapy compared to high-grade serous ovarian cancer (HGSOC).
- Defactinib works in combination with avutometinib for the treatment of recurrent KRAS mutant LGSOC. Avutometinib is a RAF/MEK clamp that induces inactive complexes of MEK with ARAF, BRAF and CRAF, potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. Defactinib is a selective FAK inhibitor that inhibits parallel pathway signaling and has demonstrated synergy with avutometinib.
- A New Drug Application (NDA) has been submitted to the U.S. Food and Drug Administration (FDA) for the avutometinib and defactinib combination for adults with recurrent KRAS mutant LGSOC, who have received at least one prior systemic therapy. The NDA for the combination was supported by data from the RAMP 201 study demonstrating a confirmed overall response rate (ORR) of 44%, a median progression free survival (PFS) of 22 months, and a disease control rate at 6 months of 70% in patients with KRAS mutant LGSOC. The NDA submission also included supportive data from the FRAME Phase 1 trial, the first study conducted with the combination in recurrent LGSOC.
- The FDA has granted Breakthrough Therapy Designation for avutometinib plus defactinib for the treatment of patients with recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC.
Development timeline for defactinib
Further information
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