Background One of the major determinants of exercise intolerance and limiting symptoms among patients with obstructive hypertrophic cardiomyopathy (HCM) is an elevated intracardiac pressure resulting from left ventricular outflow tract obstruction. Aficamten is an oral selective cardiac myosin inhibitor that reduces left ventricular outflow tract gradients by mitigating cardiac hypercontractility. Methods In this phase 3, double-blind trial, we randomly assigned adults with symptomatic obstructive HCM to receive aficamten (starting dose, 5 mg; maximum dose, 20 mg) or placebo for 24 weeks, with dose adjustment based on echocardiography results. The results for all 10 secondary end points were significantly improved with aficamten as compared with placebo. The incidence of adverse events appeared to be similar in the two groups. Conclusions Among patients with symptomatic obstructive HCM, treatment with aficamten resulted in a significantly greater improvement in peak oxygen uptake than placebo.
Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy / Maron M.S., Masri A., Nassif M.E., Barriales-Villa R., Arad M., Cardim N., Choudhury L., Claggett B., Coats C.J., Dungen H.-D., Garcia-Pavia P., Hagege A.A., Januzzi J.L., Lee M.M.Y., Lewis G.D., Ma C.-S., Michels M., Olivotto I., Oreziak A., Owens A.T., et al.. - In: THE NEW ENGLAND JOURNAL OF MEDICINE. - ISSN 0028-4793. - ELETTRONICO. - 390:(2024), pp. 1849-1861. [10.1056/NEJMoa2401424]
Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy
Masri A.;Olivotto I.;
2024
Abstract
Background One of the major determinants of exercise intolerance and limiting symptoms among patients with obstructive hypertrophic cardiomyopathy (HCM) is an elevated intracardiac pressure resulting from left ventricular outflow tract obstruction. Aficamten is an oral selective cardiac myosin inhibitor that reduces left ventricular outflow tract gradients by mitigating cardiac hypercontractility. Methods In this phase 3, double-blind trial, we randomly assigned adults with symptomatic obstructive HCM to receive aficamten (starting dose, 5 mg; maximum dose, 20 mg) or placebo for 24 weeks, with dose adjustment based on echocardiography results. The results for all 10 secondary end points were significantly improved with aficamten as compared with placebo. The incidence of adverse events appeared to be similar in the two groups. Conclusions Among patients with symptomatic obstructive HCM, treatment with aficamten resulted in a significantly greater improvement in peak oxygen uptake than placebo.
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