Avutometinib, a drug designed to target a specific pathway in cancer cells, has received orphan drug designation from the FDA for recurrent low-grade serous ovarian cancer. This designation is given to drugs with potential for treating rare diseases.
Researchers are currently evaluating a combination of Avutometinib and Defactinib in a large-scale phase 3 trial called RAMP 301. This trial aims to confirm the effectiveness and safety of the combination for patients with this type of ovarian cancer that has returned after previous treatment.
How Avutometinib Works:
Avutometinib works by blocking a key signaling pathway in cancer cells called the RAS/MAPK pathway. This pathway plays a crucial role in cell growth and division. By inhibiting this pathway, Avutometinib may be able to slow or stop the growth of cancer cells.
Phase 3 Trial Details:
The RAMP 301 trial is enrolling approximately 270 patients. Participants will be randomly assigned to receive either the combination of Avutometinib and Defactinib or a standard chemotherapy or hormonal therapy chosen by the doctor. The effectiveness of the treatment will be measured by how long patients live without their cancer progressing (progression-free survival). Other factors like overall survival, response rate, and side effects will also be monitored.
Eligibility for the Trial:
The trial is open to adults with confirmed low-grade serous ovarian cancer that has come back after at least one prior treatment. Participants must be in good overall health with adequate organ function.
Looking Ahead:
The FDA previously recognized the potential of this combination therapy by granting it breakthrough designation in 2021. The ongoing phase 3 trial aims to confirm these early findings and pave the way for potential approval and availability to patients by 2025.
Posted April 2024.
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