- Mavacamten did not meet primary endpoints in a phase III trial for nonobstructive hypertrophic cardiomyopathy.
- No new safety signals were identified, but it did not significantly improve functional capacity, symptoms, or peak oxygen consumption.
- Hypertrophic cardiomyopathy affects approximately 1 in 500 adults globally and is the most common identifiable cause of sudden cardiac death in young individuals in the United States.
- Mavacamten carries a boxed warning for the risk of heart failure.
- The trial failure reflects the challenge of demonstrating clinical benefit in less severe populations.
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