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Progression-free survival: the time a patient lives without their disease spreading or worsening.
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Application Submitted to FDA for Avutometinib Plus Defactinib in Rare Ovarian Cancer
Key Takeaways
- Avutometinib plus defactinib targets recurrent KRAS-mutant, low-grade serous ovarian cancer, a rare type with no current FDA-approved treatments.
- Avutometinib is a RAF/MEK clamp, and defactinib is a FAK inhibitor, potentially overcoming resistance mechanisms in this cancer.
The FDA received the New Drug Application for avutometinib plus defactinib as a potential treatment option for previously treated low-grade serous ovarian cancer.
The Food and Drug Administration has received an application for a new drug to treat recurrent, low-grade serous ovarian cancer, a rare disease with no current FDA-approved treatments.
A New Drug Application for avutometinib plus defactinib has been submitted to the Food and Drug Administration (FDA) for the treatment of recurrent KRAS-mutant, low-grade serous ovarian cancer previously treated with at least one systemic therapy.
According to a press release from Verastem Oncology, the drugs’ manufacturer, there are no treatments currently approved by the FDA for low-grade serous ovarian cancer, as it differs from high-grade serous ovarian cancer.
Avutometinib is an oral RAF/MEK clamp, meaning that it targets both RAF and MEK through the RAS pathway to “create a more complete and durable anti-tumor response through maximal RAS Pathway inhibition,” according to Verastem Oncology’s website. By blocking RAF and MEK in a single molecule, it may help block against tumor growth and proliferation. Defactinib is a FAK inhibitor that, according to the site, has been shown to interact well with avutometinib as a way to address the potential resistance mechanism in most patients with this disease.
“We believe that avutometinib in combination with defactinib has the potential to change the treatment paradigm for patients with recurrent KRAS-mutant, low-grade serous ovarian cancer,” Dan Paterson, president and chief executive officer of Verastem Oncology, said in the release. “Completing our [New Drug Application] submission is a significant milestone not only for Verastem as we plan for potential FDA approval in mid-2025, but also for patients, as there are no FDA-approved treatments specifically for this rare ovarian cancer.”
With the submission of a New Drug Application, the pharmaceutical company is requesting the FDA to review information about a drug — in this case, avutometinib plus defactinib — and its effectiveness to treat a disease, according to the release. The company also requested Priority Review from the FDA, meaning that, if granted, the agency will complete its review of the application within six months. During the review period, the FDA will decide whether the regimen should be approved for use within this specific disease type. The release noted that if approved, avutometinib plus defactinib will be the first treatment approved by the FDA specifically for adults with recurrent KRAS-mutant low-grade serous ovarian cancer.
This application includes updated results from the RAMP 201 study which were presented at an oncology conference in October 2024. In particular, in patients treated with avutometinib and defactinib, the confirmed response was 44% among patients with KRAS-mutant low-grade serous ovarian cancer, according to the release. Of note, the median progression-free survival was 22 months in patients with KRAS-mutant low-grade serous ovarian cancer.
According to the release, updated findings from the RAMP 201 study continue to show that treatment with avutometinib plus defactinib is well tolerated, with a 10% discontinuation rate from side effects.
Low-grade serous ovarian cancer, according to the release, is a rare ovarian cancer that is known to be highly recurrent and less sensitive to chemotherapy compared with high-grade serous ovarian cancer. An estimated 6,000 to 8,000 women live with this disease in the United States, and it is a disease that typically affects younger women. The release noted that the standard of care for low-grade serous ovarian cancer at the moment includes chemotherapy and hormone therapy.
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