Aficamten Monotherapy for Hypertrophic Cardiomyopathy

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Source: NEJM

Original: https://www.nejm.org/doi/full/10.1056/NEJMc2514558?af=R&rss=currentIssue...

Published: 2025-12-17T10:00:03Z

A study in the New England Journal of Medicine evaluated aficamten monotherapy in patients with obstructive hypertrophic cardiomyopathy (oHCM) compared to metoprolol over 24 weeks[1]. Aficamten significantly improved exercise capacity (primary endpoint was increase in peak VO2) compared to metoprolol and achieved better outcomes in symptoms and quality of life measured by the KCCQ-CSS (mean change 15.8 points vs. 8.7 points for metoprolol)[1][2]. The proportion of patients who improved by ≥1 NYHA class was 51.1% in the aficamten group versus 26.4% in the metoprolol group[1][2]. Aficamten also produced significant improvements in hemodynamic parameters, including a reduction in the left-ventricular outflow gradient and a reduction in the left atrial volume index, as well as a decrease in NT-proBNP levels[1][2][4]. Five of the six secondary endpoints were achieved in favor of aficamten; the only secondary endpoint without a significant difference was left ventricular mass index in one report[4]. The incidence of at least one treatment-emergent adverse event was similar in both groups (73.9% vs. 75.9%), with serious adverse events occurring in 8.0% of aficamten-treated patients and 6.9% of metoprolol-treated patients[1]. The authors report that the results suggest that aficamten as monotherapy provides better clinical and hemodynamic outcomes than metoprolol in symptomatic patients with oHCM[1][2][4].