Study Finds No Significant Benefits of Mavacamten in Patients with Nonobstructive Hypertrophic Cardiomyopathy
A large phase III trial reveals that mavacamten does not significantly improve symptoms or exercise capacity in patients with nonobstructive hypertrophic cardiomyopathy, highlighting the need for new therapeutic strategies.
Recent research presented at ESC Congress 2025 and published in the New England Journal of Medicine has shown that mavacamten, a cardiac myosin inhibitor, does not provide significant improvements in health status or exercise capacity for patients with symptomatic nonobstructive hypertrophic cardiomyopathy (HCM). The phase III ODYSSEY-HCM trial involved 580 adult patients across 22 countries, all experiencing symptoms consistent with nonobstructive HCM, a condition characterized by thickening of the heart muscle without significant blood flow obstruction. Participants were randomly assigned to receive either mavacamten or a placebo over a 48-week period.
The primary measures of the study were changes in patient-reported health status, evaluated by the Kansas City Cardiomyopathy Questionnaire 23-item Clinical Summary Score (KCCQ-23 CSS), and peak oxygen consumption during exercise. Results indicated no statistically significant differences between the mavacamten and placebo groups in these parameters. The average increase in quality of life scores was modest and statistically borderline, and there was no notable improvement in exercise capacity.
Safety assessments revealed that mavacamten was associated with a higher incidence of treatment-emergent adverse events, including reductions in left ventricular ejection fraction below 50%, which occurred in over 21% of the mavacamten group but in less than 2% of the placebo group. Most cases of reduced ejection fraction improved back to normal after treatment interruption.
Professor Milind Desai explained that these findings suggest mavacamten does not currently benefit patients with nonobstructive HCM, despite previous promising small-scale studies. Further analyses are underway to identify whether specific subgroups may derive benefit from this therapy. Overall, the trial emphasizes the need for ongoing research to develop effective treatments for this patient population.
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