FDA Greenlights Verastem Oncology's Combo Therapy for Rare Ovarian Cancer

The US Food and Drug Administration (FDA) has granted an accelerated approval to a significant new therapy developed by Verastem Oncology for adults suffering from a rare form of ovarian cancer. This groundbreaking oral combination therapy, known as Avmapki Fakzynja, includes avutometinib capsules and defactinib tablets. It's designed specifically for treating KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) in patients who have already undergone previous systemic treatments. This marks a notable milestone as it is the first treatment approved by the FDA for this rare disease.

LGSOC is a challenging and recurrent form of ovarian cancer, affecting between 6,000 to 8,000 women across the United States. Unlike the more common high-grade serous ovarian cancer, LGSOC is less responsive to chemotherapy. It predominantly affects younger women and offers a median survival rate of about ten years. Despite its rarity, the need for effective treatment options has been pressing.

Dan Paterson, the president and CEO of Verastem Oncology, emphasized the significance of this approval, highlighting it as the inaugural FDA-approved treatment for this specific rare cancer. He expressed pride in delivering two pioneering medicines in a single combination therapy to the LGSOC community, providing new hope to those who have long awaited effective treatments.

The FDA's approval was underpinned by the results from the mid-stage 201 clinical trial. This study involved 57 adults with measurable KRAS-mutated recurrent LGSOC. Participants received Avmapki at a dose of 3.2mg twice weekly for three weeks out of a four-week cycle, alongside Fakzynja at 200mg twice daily for the same period. This regimen continued until disease progression or until the occurrence of unacceptable toxicity. The combination therapy demonstrated a confirmed overall response rate of 44%, with responses lasting from 3.3 to 31.1 months, showcasing promising efficacy.

Under the accelerated approval program, which aims to expedite the availability of treatments for serious conditions that address unmet medical needs, the continued authorization of Avmapki Fakzynja is subject to confirmation of clinical benefits through future trials. This program plays a critical role in providing timely access to new therapies.

Nicole Andrews, chair of the STAAR Low-Grade Serous Ovarian Cancer Foundation, recognized this approval as a landmark moment for patients with this rare cancer. She expressed enthusiasm for the potential benefits of the new treatment and optimism for advancements in diagnosis, awareness, and research regarding LGSOC. The excitement within the LGSOC community reflects the hope that such developments bring in improving patient outcomes and advancing understanding of the disease.

This approval by the FDA not only represents a breakthrough in treatment options for patients with LGSOC but also underscores the ongoing commitment to advancing cancer therapies through innovation and research. It highlights the importance of continued efforts in developing effective treatments for rare and challenging diseases, providing renewed hope and possibilities for those affected.