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Aficamten Beneficial for Obstructive Hypertrophic Cardiomyopathy

Medically reviewed by Carmen Pope, BPharm. Last updated on May 16, 2024.

By Elana Gotkine HealthDay Reporter

THURSDAY, May 16, 2024 -- Treatment with the oral selective cardiac myosin inhibitor aficamten results in significantly greater improvement in peak oxygen uptake compared with placebo among patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM), according to a study published online May 13 in the New England Journal of Medicine to coincide with the European Society of Cardiology Heart Failure 2024, held from May 11 to 14 in Lisbon, Portugal.

Martin S. Maron, M.D., from the Lahey Hospital and Medical Center in Burlington, Massachusetts, and colleagues randomly assigned adults with symptomatic obstructive HCM to receive aficamten or placebo for 24 weeks (142 and 140 patients, respectively) with dose adjustment based on echocardiography results in a phase 3, double-blind trial.

The researchers found that the mean change in peak oxygen uptake was 1.8 and 0.0 mL/kg/min in the aficamten and placebo groups at 24 weeks (least-squares mean between-group difference, 1.7 mL/kg/min). For all 10 secondary end points, results improved significantly with aficamten versus placebo. In the two groups, the incidence of adverse events was similar.

"Among patients with symptomatic obstructive HCM, treatment with aficamten resulted in a significantly greater improvement in peak oxygen uptake than placebo," the authors write.

The study was funded by Cytokinetics, the manufacturer of aficamten.

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