Accelerated FDA approval marks major advance for low-grade serous ovarian cancer
The Ovarian Cancer Research Foundation (OCRF) welcomes the recent accelerated Food and Drug Administration (FDA) approval of a new combination therapy for those with recurrent low-grade serous ovarian cancer (LGSOC), an important step forward for this rare and hard-to-treat cancer.
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The combination of drugs avutometinib and defactinib (now branded as Avmapki Fakzynja), is the first targeted treatment approved for recurrent LGSOC patients whose tumours carry mutations in the KRAS gene, a subtype that has limited treatment options.
The approval was based on promising results from the RAMP 201 trial, which showed a 44% overall response rate in patients whose cancer had previously progressed after other treatments.
How do the drugs work?
Avutometinib blocks a signalling pathway that drives cancer cell growth, while defactinib targets a protein that is a key regulator of cancer cell survival and resistance; both of which are key pathways that drive tumour progression in KRAS mutated cancers.
This approval reflects years of international collaboration across industry and academic research teams, including Verastem Oncology, The Institute of Cancer Research (ICR), and the Royal Marsden NHS Foundation Trust.
The Ovarian Cancer Research Foundation is also actively investing in further research to expand future treatment options for LGSOC. A team at Peter MacCallum Cancer Centre, led by Dr Dane Cheasley, is currently investigating additional drug combinations for this disease.
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Key terms
Low-grade serous ovarian cancer: a rare type of ovarian cancer that often grows more slowly than other forms but is typically resistant to standard treatments like chemotherapy, making new treatment options especially important.
Avutometinib: A drug that blocks signals (RAF-MEK pathway) cancer cells use to grow, helping slow or stop tumour growth.
Defactinib: A drug that targets a protein called focal adhesion kinase (FAK), which cancer cells use to survive and spread.
KRAS: A gene that helps control how cells grow; when it is mutated, it can cause cancer cells to grow out of control.
Recurrence: When cancer comes back after it has been treated and after a period of no disease.
Targeted therapy: A type of treatment that works by focusing on specific changes or weaknesses in cancer cells, while aiming to spare healthy cells.
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