Verastem Oncology's NDA Accepted for Innovative LGSOC Treatment, PDUFA Date Set for 2025

Verastem Oncology Advances Cancer Treatment with FDA Acceptance of New Drug Application

Verastem Oncology, a biopharmaceutical company dedicated to developing innovative cancer therapies, announces that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for avutometinib, an oral RAF/MEK clamp, in combination with defactinib, an oral FAK inhibitor. This NDA, submitted in October 2024, seeks accelerated approval to treat adult patients with recurrent low-grade serous ovarian cancer (LGSOC) who have undergone at least one prior systemic therapy and have a KRAS mutation. The acceptance of this NDA marks a critical milestone, as there are currently no FDA-approved treatments specifically targeting this rare type of cancer.

The FDA has granted Priority Review status to Verastem’s NDA, with a Prescription Drug User Fee Act (PDUFA) action date set for June 30, 2025. Notably, the FDA has indicated that no advisory committee meeting will be held to discuss the application, streamlining the review process. Dan Paterson, president and CEO of Verastem Oncology, underscores the significance of this development, highlighting it as an essential step toward addressing a substantial unmet medical need in LGSOC. The NDA is supported by promising results from the Phase 2 RAMP 201 trial, which demonstrated a significant overall response rate and durable responses from the combination therapy. These results were prominently presented at the International Gynecologic Cancer Society Annual Global Meeting in October 2024, reinforcing the potential of this treatment.

In addition to the NDA submission, Verastem is actively enrolling patients in the ongoing RAMP 301 international Phase 3 trial, which serves as a confirmatory study for this initial indication. This underscores the company’s commitment to advancing treatment options for patients suffering from recurrent KRAS mutant LGSOC. By focusing on innovative therapies and robust clinical studies, Verastem aims to fill the gaps in treatment for this rare cancer type, thus enhancing patient outcomes and contributing to the broader oncology landscape.

In parallel with these developments, Verastem’s strategic focus on research and development highlights its commitment to addressing significant gaps in cancer treatment. The combination of avutometinib and defactinib not only targets the unique genetic profile of LGSOC but also embodies the company's dedication to precision medicine. As the company prepares for the PDUFA action date, the oncology community watches closely, recognizing the potential impact of this therapy on patient care.