Aficamten FDA Approval Status
Last updated by Judith Stewart, BPharm on May 7, 2025.
FDA Approved: No
Generic name: aficamten
Company: Cytokinetics, Incorporated
Treatment for: Hypertrophic Cardiomyopathy
Aficamten is a selective, small molecule cardiac myosin inhibitor in development for the treatment of obstructive hypertrophic cardiomyopathy.
- Hypertrophic cardiomyopathy (HCM) is a disease characterized by thickening of the cardiac muscle, which leads to the inside of the left ventricle becoming smaller and stiffer, and therefore less able to relax and fill with blood. Symptoms include chest pain, dizziness, shortness of breath, or fainting during physical activity due to limited cardiac function.
- Aficamten works in the treatment of HCM by blocking cardiac myosin, the molecular motor that drives contraction in the heart muscle. By reducing the number of active actin-myosin cross bridges during each cardiac cycle, aficamten suppresses the myocardial hypercontractility associated with HCM.
- The U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for the treatment of HCM and has assigned a revised Prescription Drug User Fee Act (PDUFA) target action date of December 26, 2025. The NDA is supported by the results from the pivotal Phase 3 clinical trial SEQUOIA-HCM which showed that treatment with aficamten for 24 weeks significantly improved exercise capacity compared to placebo.
- The FDA previously granted aficamten Orphan Drug Designation for the treatment of symptomatic HCM in January 2021 and Breakthrough Therapy Designation for the treatment of obstructive HCM in December 2021.
Development timeline for aficamten
Further information
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