Mavacamten: a first-in-class myosin inhibitor for obstructive hypertrophic cardiomyopathy.

ABSTRACT: Mavacamten is a first-in-class, targeted, cardiac-specific myosin inhibitor approved by the US Food and Drug Administration for the treatment of adults with symptomatic New York Heart Association Classes II and III obstructive hypertrophic cardiomyopathy (oHCM). Mavacamten was developed to target the hyper-contractile phenotype, which plays a critical role in the pathophysiology of the disease. In Phase 2 and 3 clinical trials, mavacamten was well tolerated, reduced left ventricular outflow tract gradients, improved exercise capacity and symptoms, and was associated with improvements in other clinically relevant parameters, such as patient-reported outcomes and circulating biomarkers. In addition, treatment with mavacamten was associated with evidence of favourable cardiac remodelling in m

SUBMITTER: Braunwald E 

PROVIDER: S-EPMC10659958 | biostudies-literature | 2023 Nov

REPOSITORIES: biostudies-literature