May 8, 2025 – The U.S. Food and Drug Administration (FDA) approved Verastem Oncology’s combination therapy, branded as AVMAPKI™ FAKZYNJA™ Co-pack, for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.
AVMAPKI™ FAKZYNJA™ becomes the first and only drug approved by FDA for treating adults with LGSOC that involves a mutation in the KRAS gene. Its approval will give hope to thousands of women living with this disease.
The combination drug (AVMAPKI™ FAKZYNJA™) works by blocking multiple pathways that promote cancer cell survival and growth. AVMAPKI™ FAKZYNJA’s accelerated approval is based on the Phase 2 RAMP 201 clinical trial, which assessed the combination of FAKZYNJA and AVMAPKI in adult patients with measurable KRAS-mutated recurrent LGSOC.
Views and Statements
Verastem reports that around 80,000 women worldwide are living with low-grade serous ovarian cancer (LGSOC). The company added that the new U.S. approval leads to an initial opportunity of reaching around 1800 patients. LGSOC disproportionately affects younger women and has poor response rates to chemotherapy.
During the trial, AVMAPKI™ FAKZYNJA™ significantly improved patient’s response rate without any serious side effects. This positions the therapy as a leading contender in addressing the global burden of LGSOC.
Dan Paterson, president and CEO of Verastem Oncology, stated, “Approval of AVMAPKI™ FAKZYNJA™ for patients with KRAS-mutated recurrent LGSOC represents the first-ever FDA-approved treatment for this rare cancer. It also brings a new ray of hope for people living with this disease who are desperately seeking new and effective treatment options.” He further added, “The company is proud to bring two new medicines in one combination treatment to the LGSOC patients.”
In the words of Nicole Andrews, chair of the STAAR Low-Grade Serous Ovarian Cancer Foundation, “Today marks a major milestone with the first-ever FDA-approved treatment options specifically for patients with recurrent LGSOC with a KRAS mutation. This breakthrough brings new hope to the LGSOC community.”
Impact and Future Outlook
Prior to this approval, there were no FDA-approved treatments for KRAS-mutated recurrent LGSOC. However, the approval of AVMAPKI™ FAKZYNJA™ changed this for once and all by providing a targeted therapy option that specifically addresses the KRAS mutation. This approval marks a significant breakthrough in the treatment landscape for recurrent low-grade serous ovarian cancer (LGSOC).
Development and approval of ovarian cancer drugs like AVMAPKI™ FAKZYNJA not only fills a longstanding therapeutic gap but also offers new hope for patients who previously had limited or no effective treatment options. Regulatory success of AVMAPKI™ FAKZYNJA™ Co-pack may encourage other companies to increase their investment in R&D for developing advanced ovarian cancer drugs solutions.
Sources:
News Outlet: Reuters
U.S. Federal Agency: FDA