After a significant regulatory notification, shares of Verastem, Inc. (NASDAQ: VSTM) are up 39.51% to $5.12 as of the recent check on the US stock charts. VSTM’s New Drug Application (NDA) for avutometinib in combination with defactinib has been approved by the US Food and Drug Administration (FDA) via the accelerated approval process.
Innovative Therapy for Uncommon Ovarian Cancer
The suggested treatment is intended for adult patients with recurrent low-grade serous ovarian cancer (LGSOC) who have KRAS mutations. It consists of the oral RAF/MEK clamp avutometinib and the oral FAK inhibitor defactinib. The Prescription Drug User Fee Act or PDUFA action date is planned for June 30, 2025, as a result of the NDA’s October 2024 submission being awarded Priority Review.
For this study, the FDA does not presently have any plans to convene an advisory committee meeting. A major advancement in oncology, if approved, the medicine would be the first FDA-approved treatment especially for recurrent KRAS-mutant LGSOC.
Clinical Evidence Supporting the Filing
The NDA builds on compelling evidence from the Phase 2 RAMP 201 trial, where the avutometinib-defactinib combination demonstrated a robust overall response rate, confirmed by an independent central review.
These responses were often durable, with patients generally tolerating the treatment well. Supporting data from the FRAME Phase 1 trial further substantiates the efficacy and safety of this innovative approach.
Pathway to Expanded Treatment Options
In parallel with the FDA review process, Verastem is advancing its RAMP 301 Phase 3 trial, enrolling patients with recurrent LGSOC regardless of KRAS mutation status. This confirmatory trial aims to broaden the therapy’s indication, potentially addressing a wider spectrum of patients.
With the FDA’s Priority Review designation, Verastem is positioned to deliver a groundbreaking treatment addressing a critical gap in cancer care. VSTM is actively preparing for a commercial launch anticipated in mid-2025, signaling hope for patients battling this rare disease.
