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FDA Accelerated Approval of Avmapki Fakzynja Co-pack for KRAS-Mutated LGSOC
On May 8, 2025, the Food and Drug Administration approved the use of avutometinib and defactinib (Avmapki Fakzynja Co-pack, Verastem, Inc.) for adults with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have already received treatment before.
The effectiveness was evaluated in RAMP-201 (NCT04625270), a study that included 57 adult patients with measurable KRAS-mutated recurrent low-grade serous ovarian cancer. Patients were mandated to have undergone a minimum of one previous systemic therapy, which included a platinum-based regimen. The KRAS mutation status was established using a prospective local analysis of tumor samples. Patients were administered avutometinib 3.2 mg orally biweekly (on Day 1 and Day 4) and defactinib 200 mg orally twice daily for the initial 3 weeks of each 4-week cycle, unless disease progression or intolerable toxicity occurred.
RAMP-201 Clinical Trial: Superior Response and Survival Data
The primary effectiveness endpoint was the overall response rate (ORR), evaluated by a blinded independent review committee in accordance with RECIST v1.1. An extra efficacy outcome measure was the duration of response (DOR). The confirmed overall response rate (ORR) was 44% (95% confidence interval: 31, 58), and the duration of response (DOR) ranged from 3.3 months to 31.1 months.
Safety Profile and Managing Side Effects
The most prevalent adverse reactions (≥25%), including laboratory abnormalities, comprised elevated creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, reduced hemoglobin, elevated alanine aminotransferase, vomiting, increased blood bilirubin, elevated triglycerides, diminished lymphocyte count, abdominal pain, dyspepsia, acneiform dermatitis, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, xerosis, dyspnea, cough, urinary tract infection, and diminished neutrophil count.
Recommended Dosage and Administration
The recommended dose of avutometinib is 3.2 mg, which is taken as four 0.8 mg capsules by mouth every two weeks (on Day 1 and Day 4) for the first 3 weeks of each 4-week cycle, and this continues until the disease gets worse or side effects become too severe. The recommended dose of defactinib is 200 mg (one tablet) taken by mouth two times a day for the first 3 weeks of each 4-week cycle, and this continues until the disease gets worse or side effects become too severe.
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About Susan Hau
Susan Hau is a distinguished researcher in the field of cancer cell therapy, with a particular focus on T cell-based approaches and cancer vaccines. Her work spans several innovative treatment modalities, including CAR T-cell therapy, TIL (Tumor-Infiltrating Lymphocyte) therapy, and NK (Natural Killer) cell therapy. Hau's expertise lies in cancer cell biolo…
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This article is for educational purposes only and should not replace medical advice, diagnosis, or treatment from a qualified oncology specialist. Every patient's case is different. Treatment decisions should always be made after a review of complete medical records by the treating medical team.
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