Verastem receives Orphan Drug Designation from FDA for avutometinib

Verastem Oncology announced that the FDA granted Orphan Drug Designation, or 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, or LGSOC. Approximately 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.