Verastem Oncology Receives FDA Orphan Drug Designation for Avutometinib and Defactinib for the Treatment of Pancreatic Cancer
Verastem Oncology, a biopharmaceutical company committed to advancing new medicines for patients with cancer, announced that it has initiated the rolling submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) seeking accelerated approval of the combination of avutometinib, a RAF/MEK clamp, and defactinib, a selective FAK inhibitor, for adult patients with recurrent KRAS mutant (KRAS mt) low-grade serous ovarian cancer (LGSOC), who received at least one prior systemic therapy. The rolling review process allows the company to submit completed sections of an application for review by the FDA before all sections become available. The initial sections of the application will include the nonclinical and quality sections. In discussions with the FDA, Verastem reached agreement to submit a primary efficacy analysis based on the RAMP 201 study with 12 months of follow up. Based on discussions with the FDA, we understand that the proposed indication for final submission of the clinical module can be expanded in the event Verastem provides data that demonstrates a substantial improvement over available therapy in the KRAS wild-type (KRAS wt) population. FDA has accepted Verastem’s plan to submit the clinical module in the second half of 2024 to complete the NDA application. Previously, the FDA granted Breakthrough Therapy Designation (BTD) for the combination for treatment of patients with recurrent LGSOC, regardless of KRAS status, following one or more previous lines of therapy and Orphan Drug Designation (ODD) for the combination in certain LGSOC indications. The company plans to request a priority review of the NDA. Currently, there are no FDA-approved treatments specifically for recurrent LGSOC.
Verastem Oncology Announces the Initiation of a Rolling Submission of NDA to FDA Seeking Accelerated Approval of Avutometinib and Defactinib Combination for the Treatment of Adult Patients with Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer
Verastem Receives Orphan Drug Designation from FDA for Avutometinib Alone
Verastem Oncology Outlines Key 2024 Strategic Priorities
Verastem Announces Initiation of a Confirmatory Phase 3 Trial of Avutometinib
Verastem Oncology Announces Efficacy and Safety Data of Avutometinib
Verastem Presents Avutometinib and Defactinib Combination Program Updates at the 5th Annual RAS-Targeted Drug Development Summit
BOSTON--(BUSINESS WIRE)--Verastem Oncology (Nasdaq: VSTM) (the “Company”), a biopharmaceutical company committed to advancing new medicines for patients with cancer, announced today that it has finalized with the U.S. Food and Drug Administration (FDA) the design of its confirmatory Phase 3 trial to evaluate the combination of avutometinib and defactinib for the treatment of recurrent low-grade serous ovarian cancer (LGSOC). RAMP 301, a randomized global confirmatory trial, will evaluate the efficacy and safety of avutometinib and defactinib versus standard of care (SOC) chemotherapy and hormonal therapy in patients with recurrent LGSOC. RAMP 301 is expected to begin enrollment in the second half of this year.
The RAF/MEK and FAK inhibitor combination avutometinib (VS-6766) plus defactinib (VS-6063) represents a novel area of exploration that has displayed clinical activity and a manageable safety profile in patients with low-grade serous ovarian cancer, according to Bradley J. Monk, MD, FACS, FACOG.