The FDA has approved aficamten for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), with the aim of improving functional capacity and symptoms.
Aficamten is an allosteric, reversible inhibitor of cardiac myosin, which reduces cardiac contractility and left ventricular outflow tract (LVOT) obstruction, a key pathophysiological feature of oHCM. Aficamten has been approved as 5mg, 10mg, 15mg and 20mg tablets, with a label allowing flexible dosing and no requirement for routine drug–drug interaction monitoring.
The approval comes following the results from the pivotal phase 3 SEQUOIA-HCM trial (NCT05186818), published in the New England Journal of Medicine. Over 24 weeks, aficamten significantly improved exercise capacity compared to placebo, peak oxygen uptake measured by cardiopulmonary exercise testing increased by 1.8 mL/kg/min. Improvements were consistent across prespecified subgroups, including age, sex and background beta-blocker use. Reductions in symptoms were also reported.
In the trial, aficamten was generally well tolerated. Serious treatment-emergent adverse events occurred in 5.6% of patients receiving aficamten and 9.3% of those receiving placebo. Reductions in left ventricular ejection fraction below 50% were observed in a small proportion of patients receiving aficamten. Hypertension was the only adverse event reported in more than 5% of patients and occurred more frequently with aficamten than with placebo.
The US prescribing information includes a boxed warning for the risk of heart failure due to systolic dysfunction. Echocardiographic monitoring of systolic dysfunction is required before and during treatment, and aficamten will be available only through a Risk Evaluation and Mitigation Strategy (REMS) programme.
Speaking on the approval, Lisa Salberg, Founder and CEO of the Hypertrophic Cardiomyopathy Association (HCMA), said: “For far too long, we’ve had few options to address our needs, and the approval of Myqorzo is a long-awaited and major addition to bring new hope to patients living with oHCM.“
Reference:
Maron MS, Masri A, Nassif ME, et al. Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. 2024;390:1849–1861.
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Cite: Aficamten secures FDA approval for symptomatic obstructive hypertrophic cardiomyopathy. touchCARDIO. 22 December 2025.
Editor: Victoria Smith, Senior Content Editor.
Disclosures: This article was created by the touchCARDIO team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
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