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Hypertrophic cardiomyopathy (HCM) is a complex cardiac disease with highly variable phenotypic expression and clinical course most often caused by sarcomeric gene mutations resulting in left ventricular hypertrophy, fibrosis, hypercontractility, and diastolic dysfunction. For almost 60 years, HCM has remained an orphan disease and still lacks a disease-specific treatment. This review summarizes recent preclinical and clinical trials with repurposed drugs and new emerging pharmacological and gene-based therapies for the treatment of HCM. The off-label drugs routinely used alleviate symptoms but do not target the core pathophysiology of HCM or prevent or revert the phenotype. Recent advances in the genetics and pathophysiology of HCM led to the development of cardiac myosin adenosine triphosphatase inhibitors specifically directed to counteract the hypercontractility associated with HCM-causing mutations. Mavacamten, the first drug specifically developed for HCM successfully tested in a phase 3 trial, represents the major advance for the treatment of HCM. This opens new horizons for the development of novel drugs targeting HCM molecular substrates which hopefully modify the natural history of the disease. The role of current drugs in development and genetic-based approaches for the treatment of HCM are also discussed.

Hypertrophic cardiomyopathy: an up-to-date snapshot of the clinical drug development pipeline