Press release
Verastem Oncology Receives Orphan Drug Designation from FDA for Avutometinib Alone or in Combination With Defactinib in Recurrent Low-Grade Serous Ovarian Cancer
Recurrent low-grade serous ovarian cancer is a rare cancer with no FDA-approved treatments Ongoing Phase 3 RAMP 301 trial is evaluating avutometinib and
About this update from Verastem, Inc.
[{"type":"text","content":"\nRecurrent low-grade serous ovarian cancer is a rare cancer with no FDA-approved treatments\n\n\nOngoing Phase 3 RAMP 301 trial is evaluating avutometinib and defactinib in recurrent low-grade serous ovarian cancer\n\n\nOn track to submit rolling NDA for Accelerated Approval in H1 2024\n\n\n BOSTON--(BUSINESS WIRE)--\nVerastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to avutometinib, a RAF/MEK clamp, alone or in combination with defactinib, a selective FAK inhibitor, for the treatment of all patients with recurrent low-grade serous ovarian cancer (LGSOC).\n\n\n“The FDA Orphan Drug Designation for avutometinib alone or in combination with defactinib in low-grade serous ovarian cancer is an important step in recognizing this rare cancer as a distinct disease that currently has no FDA-approved treatments,” said Dan Paterson, president and chief executive officer of Verastem Oncology. “We are rapidly advancing the development program for avutometinib and defactinib in low-grade serous ovarian cancer with our ongoing Phase 3 clinical trial to deliver this new combination treatment to patients as quickly as possible. We remain on track to begin submission of an NDA to the FDA for Accelerated Approval of this combination in the first half of 2024 and preparing for a potential launch in 2025.”\n\n\nApproximately 70% of LGSOC is associated with RAS/MAPK Pathway alterations including KRAS, NRAS HRAS, and other non-RAS associated mutations. Unfortunately, approximately 85% of patients with LGSOC experience recurrence of the disease. LGSOC is a highly recurrent, chemotherapy-resistant cancer, associated with slow tumor growth and high mortality rate. An estimated 6,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects a younger patient population with bimodal peaks at ages 20-30 and 50-60 years of age and has a median survival of approximately ten years. The majority of patients experience severe pain and complications as the disease progresses. While chemotherapy is the standard of care for this disease, there are no treatments specifically approved by the FDA to treat LGSOC.\n\n\nAbout the Avutometinib and Defactinib Combination\n\n\nAvutomet...