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Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy
Milind Y Desai1,2, Anjali T Owens3, Theodore Abraham4
1Hypertrophic Cardiomyopathy Center, Heart, Vascular, and Thoracic Institute, Cleveland Clinic, Cleveland.
The New England Journal of Medicine
|September 1, 2025
Summary
Mavacamten did not significantly improve exercise capacity or patient-reported health in nonobstructive hypertrophic cardiomyopathy (HCM). Further research is needed to understand its role in this patient population.
Area of Science:
- Cardiology
- Pharmacology
- Clinical Trials
Background:
- Mavacamten is approved for symptomatic obstructive hypertrophic cardiomyopathy (HCM).
- Its efficacy in nonobstructive HCM is not well-established.
- This study investigates mavacamten's effects in patients with symptomatic nonobstructive HCM.
Purpose of the Study:
- To evaluate the efficacy of mavacamten in improving functional capacity.
- To assess the impact of mavacamten on patient-reported health status in nonobstructive HCM.
- To determine if mavacamten offers benefits beyond placebo in this patient group.
Main Methods:
- A Phase 3, international, double-blind, placebo-controlled trial.
- 289 patients received mavacamten, 291 received placebo for 48 weeks.
- Primary endpoints: change in peak oxygen uptake and Kansas City Cardiomyopathy Questionnaire clinical summary score (KCCQ-CSS).
Main Results:
- Mavacamten showed a non-significant trend towards improved peak oxygen uptake (0.47 ml/kg/min difference, P=0.07).
- KCCQ-CSS scores improved slightly more with mavacamten (2.7 points difference, P=0.06), but not significantly.
- Adverse events, including reduced ejection fraction, were more frequent with mavacamten.
Conclusions:
- Mavacamten did not demonstrate statistically significant improvements in functional capacity or symptoms compared to placebo in nonobstructive HCM.
- The drug did not meet primary endpoints in this study population.
- Further investigation may be warranted to clarify mavacamten's role in nonobstructive HCM.
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