Verastem Oncology has received the US Food and Drug Administration (FDA) approval for its Avmapki Fakzynja Co-Pack for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC).
Avutometinib inhibits MEK kinase activity and blocks compensatory reactivation by upstream RAF, both regulators of the RAS/RAF/MEK/ERK (MAPK) pathway.
Defactinib is an FAK inhibitor that complements avutometinib by providing effective blockade of signalling pathways that drive tumour growth and drug resistance in RAS/MAPK pathway-dependent cancers.
The combination therapy, comprising avutometinib capsules and defactinib tablets, was approved under the FDA’s accelerated approval programme.
A continued approval is expected contingent upon further verification of clinical benefits in a confirmatory trial.
The Avmapki Fakzynja Co-Pack is the world’s first treatment approved for KRAS-mutated recurrent LGSOC, a cancer distinct from high-grade serous ovarian cancer.
Verastem Oncology president and CEO Dan Paterson said: “Today’s approval of Avmapki Fakzynja Co-Pack for patients with KRAS-mutated recurrent low-grade serous ovarian cancer represents not only the first-ever FDA-approved treatment specifically for this rare cancer but also a new day for people living with this disease who have been in desperate need of new treatment options.
“We are very proud to bring two innovative medicines in one combination treatment to the LGSOC community.
“We thank the researchers, patients, and their families participating in our clinical trials, the patient advocacy community, the FDA, and everyone at Verastem for their dedication and commitment to helping us bring Avmapki Fakzynja Co-Pack to patients in the US.”
The FDA approval of Avmapki Fakzynja Co-Pack is based on the Phase 2 RAMP 201 study, which showed a 44% overall response rate.
The decision came ahead of the FDA’s scheduled Prescription Drug User Fee Act (PDUFA) action date of 30 June 2025.
The agency granted Priority Review and Breakthrough Therapy Designation for the treatment after prior therapy, including platinum-based chemotherapy, in May 2021.
In addition, avutometinib, alone or in combination with defactinib, received FDA Orphan Drug Designation for LGSOC treatment in July last year.
Verastem plans to commercialise Avmapki Fakzynja Co-Pack in the US as a convenient oral combination, co-packaged together.
