FDA Approves AVMAPKI™ FAKZYNJA™ as First Treatment for KRAS-Mutated Recurrent Low-Grade Serous Ovarian Cancer

Verastem Oncology, a biopharmaceutical firm specializing in treatments for cancers driven by the RAS/MAPK pathway, has announced a significant milestone. The U.S. Food and Drug Administration (FDA) has granted accelerated approval to the AVMAPKI™ FAKZYNJA™ CO-PACK, an oral combination therapy designed for adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have undergone prior systemic treatment. This approval represents the first-ever FDA-sanctioned medication specifically for this rare type of ovarian cancer.

The approval of AVMAPKI FAKZYNJA CO-PACK was largely influenced by results from the Phase 2 RAMP 201 study, which demonstrated a tumor response rate of 44%. Verastem's president and CEO, Dan Paterson, expressed enthusiasm about the approval, highlighting the urgent need for new treatment options for patients suffering from this rare cancer. The combination of AVMAPKI and FAKZYNJA offers a promising new therapeutic approach for individuals with this specific genetic mutation in their tumors.

Low-grade serous ovarian cancer is a rare and recurring form of cancer that does not respond well to conventional chemotherapy. It is different from more common high-grade serous ovarian cancers and typically affects younger women. With approximately 6,000 to 8,000 U.S. cases and 80,000 worldwide, the need for targeted therapies is critical. This new treatment provides hope and a new standard of care for women facing this challenging diagnosis.

Research behind the approval included the RAMP 201 trial, which assessed the efficacy of the combination of avutometinib (AVMAPKI) and defactinib (FAKZYNJA) in adults with measurable KRAS-mutated recurrent LGSOC. During the study, participants received AVMAPKI and FAKZYNJA over a four-week cycle, and the overall response rate was a key measure of success. The trial revealed that 44% of patients experienced a significant tumor shrinkage, with the median duration of response ranging from 3.3 to 31.1 months.

The safety profile of the AVMAPKI FAKZYNJA CO-PACK was also evaluated, with findings indicating some possible serious side effects, such as eye disorders, skin toxicities, and liver issues. Common side effects reported included increased enzyme levels in the blood, nausea, fatigue, and skin rashes. Nevertheless, the therapy was generally well-tolerated, and the potential benefits appear to outweigh the risks for many patients.

Verastem Oncology is committed to supporting patients through its Verastem Cares™ program, which offers assistance with access and reimbursement for those prescribed this new therapy. The program is designed to ease the burden on patients and ensure they can receive the necessary treatment.

The company is also planning further trials to explore additional uses of AVMAPKI and FAKZYNJA in treating RAS/MAPK-driven tumors. These studies will focus on a variety of cancer types, including non-small cell lung cancer and pancreatic cancer.

The approval of AVMAPKI FAKZYNJA CO-PACK marks a significant advancement in the treatment landscape for low-grade serous ovarian cancer, offering new hope for patients and opening up a new avenue for cancer therapy. This milestone is celebrated by patients, researchers, and clinicians alike, who have long awaited an effective treatment for this difficult-to-treat cancer.