FDA Approves First Targeted Treatment for KRAS-Mutated Low-Grade Serous Ovarian Cancer

FDA Approves First-Ever Treatment for Recurrent Low-Grade Serous Ovarian Cancer (LGSOC)

5/17/2025

Accelerated approval of avutometinib and defactinib offers new hope for U.S. patients with KRAS-mutated LGSOC

In a landmark decision, the U.S. Food and Drug Administration (FDA) has granted accelerated approval for the first-ever treatment developed specifically for recurrent low-grade serous ovarian cancer (LGSOC).

The newly approved combination— avutometinib and defactinib —will be available in the United States under the commercial names AVMAPKI™ and FAKZYNJA™, co-packaged by Verastem Oncology. This treatment is approved for adult patients with recurrent LGSOC who have a KRAS mutation—a genetic change found in 30% of LGSOC cases.

“One of the most devastating aspects of my LGSOC diagnosis was learning there were no FDA-approved treatments specifically for this rare cancer,” said Nicole Andrews, Chair of the STAAR Low-Grade Serous Ovarian Cancer Foundation. “Today, we're celebrating a milestone. The low-grade serous ovarian cancer community is hopeful and excited about this progress toward better treatment, awareness, and research.”

What Is a KRAS Mutation?

KRAS is a gene that helps control how cells grow. When this gene is mutated, it can send signals that tell cancer cells to grow and spread. These mutations are common in several types of cancer, including LGSOC. Until now, there have been no FDA-approved treatments that specifically target KRAS-mutated low-grade serous ovarian cancer.

How Do Avutometinib and Defactinib Work?

These two medicines target different pathways involved in cancer cell survival:

Avutometinib blocks part of the MAPK pathway, which KRAS mutations often rely on to promote cancer growth.
Defactinib targets a protein called FAK, which helps cancer cells stick together and avoid dying.

Together, they work to interrupt the signals that help cancer cells survive and spread. In clinical trials, this combination showed promise in helping shrink or control tumors in some patients with KRAS-mutated LGSOC.

What Is Accelerated Approval?

The FDA’s accelerated approval program allows promising treatments to reach patients more quickly, especially when there are no existing approved options. This approval is based on data from clinical trials showing strong early results. A larger confirmatory study— RAMP 301 —is already underway to gather more data about long-term benefit and to evaluate the treatment in patients without a KRAS mutation.

What This Means for U.S. Patients

If you’re a U.S.-based patient with recurrent LGSOC and have tested positive for a KRAS mutation, this new treatment could now be an option for you. You’ll need to speak with your oncologist about:

Whether you’ve been tested for KRAS mutations (and how to get tested if not).
Whether this treatment might be appropriate for your case.
How to access the medications and what insurance coverage may look like.

This approval only applies in the United States, and only for KRAS-mutated cases. But it marks a major step forward—and a validation of years of research, advocacy, and patient participation in clinical trials.

Low-grade serous ovarian cancer is distinct from more common ovarian cancers. It tends to grow slowly but resists standard chemotherapy, leaving patients with few effective options. The approval of AVMAPKI and FAKZYNJA is a turning point in the treatment landscape—one that was made possible through research, advocacy, and patient participation in clinical trials.

Learn more about the FDA approval

Read Verastem’s full announcement here:
FDA Approves AVMAPKI and FAKZYNJA for KRAS-Mutated LGSOC

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FDA Approves First-Ever Treatment for Recurrent Low-Grade Serous Ovarian Cancer (LGSOC)

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