Martin S Maron, Ahmad Masri, Michael E Nassif, Roberto Barriales-Villa, Michael Arad, Nuno Cardim, Lubna Choudhury, Brian Claggett, Caroline J Coats, Hans-Dirk Düngen, Pablo Garcia-Pavia, Albert A Hagège, James L Januzzi, Matthew M Y Lee, Gregory D Lewis, Chang-Sheng Ma, Michelle Michels, Iacopo Olivotto, Artur Oreziak, Anjali T Owens, John A Spertus, Scott D Solomon, Jacob Tfelt-Hansen, Marion van Sinttruije, Josef Veselka, Hugh Watkins, Daniel L Jacoby, Stephen B Heitner, Stuart Kupfer, Fady I Malik, Lisa Meng, Amy Wohltman, Theodore P Abraham
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...
May 13, 2024: New England Journal of Medicine