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MUNICH — In patients with symptomatic nonobstructive hypertrophic cardiomyopathy, aficamten led to greater improvements in health status and exercise capacity at 36 weeks compared with placebo, according to data from the ACACIA-HCM trial.
Aficamten (Myqorzo, Cytokinetics) is the first cardiac myosin inhibitor to show benefit in patients with nonobstructive HCM. In December 2025, the FDA approved aficamten to improve heart failure symptoms and exercise capacity in adults with symptomatic obstructive HCM. There are currently no proven treatment options for nonobstructive HCM beyond heart transplantation.
“Nonobstructive HCM is a common heart disease. Patients experience debilitating symptoms with exertion that limits their exercise capacity,” Ahmad Masri, MD, MS, cardiomyopathy section head and associate professor of medicine in the division of cardiovascular medicine at Oregon Health & Science University, said during a press conference at the European Society of Cardiology Congress.
The phase 3, double-blind ACACIA-HCM trial included 517 adults with symptomatic nonobstructive HCM (mean age, 55 years; 54% women; mean left ventricular ejection fraction, 68%) who were randomly assigned to treatment with aficamten or placebo for up to 72 weeks. The starting dose was 5 mg and the maximum dose was 20 mg. The dose was escalated if LVEF was 60% or higher and deescalated if less than 50%.
