Ovarian Cancer on White Background illustration
Pune, India | August 21, 2025– The U.S. Food and Drug Administration (FDA) has granted accelerated approval to a new combination therapy for adults with recurrent, KRAS-mutated low-grade serous ovarian cancer who have previously received systemic treatment. This dual-drug regimen includes avutometinib and defactinib, two kinase inhibitors that work together to slow tumor growth.
Marketed as Avmapki Fakzynja Co-Pack, this approval represents a meaningful step forward in precision oncology. It delivers a targeted option for patients with limited existing treatments.
The decision follows results from the RAMP-201 clinical trial, which enrolled 57 patients with measurable disease. Participants took avutometinib twice weekly and defactinib twice daily in three-week treatment cycles, followed by one week off. The trial reported an overall response rate (ORR) of 44%. Some patients maintained their response for more than 31 months.
These results are especially significant given the rarity of this cancer subtype and the lack of approved therapies targeting KRAS mutations in ovarian cancer.
To speed up access, the FDA employed its Real-Time Oncology Review (RTOR) and Assessment Aid programs. The therapy also received breakthrough therapy, orphan drug, and priority review designations, signaling its potential to address a serious unmet medical need.
While the benefits are clear, the treatment can lead to side effects. Common reactions include nausea, fatigue, skin rash, vision issues, and elevated liver enzymes or creatine phosphokinase.
Physicians should monitor patients closely and educate them about these possible adverse effects. Regular evaluations can help manage risks and maintain treatment safety.
As part of the conditional approval, Verastem—the drug’s manufacturer—must complete a Phase 3 confirmatory trial. Named RAMP-301, the trial is expected to conclude by June 2028. It will compare the avutometinib–defactinib combination to physician-selected standard treatments.
Key objectives include measuring progression-free survival and overall survival, while also confirming KRAS mutation status through centralized testing. These efforts will help determine the long-term effectiveness and safety of the therapy.
The FDA’s decision illustrates its commitment to balancing rapid access with scientific rigor. Accelerated approval allows patients to benefit from promising treatments sooner, while requiring continued evidence collection to support long-term use.
The approval of Avmapki Fakzynja is more than just a regulatory success. It brings new hope for patients with a rare, difficult-to-treat form of ovarian cancer defined by a specific molecular signature.
As RAMP-301 moves forward, oncologists and researchers will track its progress closely. If the results validate current findings, the combination could become a standard therapy for KRAS-mutated ovarian cancer.
In the meantime, healthcare professionals should remain alert to potential safety issues and report adverse events through the FDA’s MedWatch system. This helps maintain a high level of post-approval oversight.
This milestone reflects the FDA’s broader focus on advancing precision medicine. Through flexible yet robust approval pathways, the agency supports timely access to novel treatments while safeguarding public health.
For patients confronting a tough diagnosis, this new treatment offers a renewed sense of possibility—and marks another advance in the evolving field of targeted cancer therapies.
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