- Question
- Does mavacamten, a selective cardiac myosin inhibitor, improve exercise capacity and symptoms in patients with symptomatic obstructive hypertrophic cardiomyopathy?
- Design
- Phase 3, randomised (1:1), double-blind, placebo-controlled trial conducted at 68 cardiovascular centres in 13 countries, with 251 patients and 30 weeks of treatment.
- Population
- Adults with hypertrophic cardiomyopathy, a left ventricular outflow tract gradient of 50 mm Hg or greater, and NYHA class II-III symptoms.
- Intervention
- Mavacamten, starting at 5 mg, for 30 weeks.
- Control
- Placebo for 30 weeks.
- Primary outcome
- Composite clinical response: a 1.5 mL/kg per min or greater increase in peak oxygen consumption (pVO2) with at least one NYHA class reduction, or a 3.0 mL/kg per min or greater pVO2 increase without NYHA class worsening.
- Results
- The primary endpoint was met by 45 of 123 patients (37%) on mavacamten versus 22 of 128 (17%) on placebo (difference +19.4%, 95% CI 8.7 to 30.1; p=0.0005). Post-exercise LVOT gradient fell by 36 mm Hg more than with placebo (p<0.0001).
- Safety
- Safety and tolerability were similar to placebo, and treatment-emergent adverse events were generally mild. One sudden death occurred, in the placebo group.
- Conclusion
- Mavacamten improved exercise capacity, LVOT obstruction, functional class, and health status in obstructive hypertrophic cardiomyopathy.
- Why it matters
- This pivotal trial showed the benefit of a disease-specific treatment targeting the underlying pathophysiology, paving the way for cardiac myosin inhibitors as a targeted therapeutic option.
Card generated automatically with AI from the abstract of the original publication and the trial registry; every figure was checked against those sources. It may contain errors: verify the data at the source before using it for clinical decisions.
Generated on October 14, 2026