Randomized Clinical Trial: Aficamten improves peak oxygen uptake in symptomatic obstructive hypertrophic cardiomyopathy – N Engl J Med

Study Design and Population: This phase 3, double-blind trial randomized 282 adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM) to receive aficamten or placebo for 24 weeks. The primary outcome was the change in peak oxygen uptake, with secondary outcomes including changes in the Kansas City Cardiomyopathy Questionnaire score and New York Heart Association functional class.

Main Findings: Aficamten significantly increased peak oxygen uptake by 1.7 ml/kg/min (95% CI, 1.0 to 2.4; P<0.001) compared to placebo. Improvements were also seen in all secondary outcomes. The incidence of adverse events was similar between the groups.

Implications for Practice: Aficamten shows promise in improving exercise capacity and symptoms in patients with obstructive HCM, potentially offering a new therapeutic option. Further research may confirm its long-term benefits and safety.

Reference (link to abstract – $ for full-text):

Maron, M. S., et al. (2024). Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy. New England Journal of Medicine. DOI: 10.1056/NEJMoa2401424.