Key result
Mavacamten emerges as the first new pharmacological therapy for HCM in ~35 years.
Why the study?
Following the phase 3 EXPLORER-HCM trial, perspective was needed on the potential role of the novel myosin inhibitor mavacamten compared with established therapies for obstructive HCM.
This article provides perspective on the potential role of mavacamten compared with established therapies for obstructive hypertrophic cardiomyopathy.
May warrant selective use in obstructive HCM pending head-to-head data; leaves open optimal positioning versus established therapies.
he application of contemporary treatment strategies to hypertrophic cardiomyopathy (HCM) has transformed this common genetic heart disease from one of grim progression to one now compatible with normal longevity and excellent quality of life. Mavacamten, a myosin inhibitor, represents the first pharmacological strategy advanced for HCM in nearly 35 years, culminating recently with publication of a phase 3 randomized clinical trial (EXPLORER-HCM [Mavacamten for Treatment of Symptomatic Obstructive Hypertrophic Cardiomyopathy]). 1 Given the enthusiasm surrounding this novel drug, even before Food and Drug Administration approval, it is timely to provide perspective on the potential role for mavacamten, particularly compared with the current established therapies for patients with obstructive HCM.
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Maron et al. (2021) conducted a review in Obstructive hypertrophic cardiomyopathy. Mavacamten vs. Established therapies was evaluated. Mavacamten, a novel myosin inhibitor, represents the first new pharmacological strategy for hypertrophic cardiomyopathy in nearly 35 years, prompting a review of its role versus established therapies.
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