Mavacamten provides sustained improvement of HOCM symptoms even beyond 1 year: VALOR-HCM trial

The first drug in class of cardiac myosin inhibitors , mavacamten, received US FDA approval in 2022 following results of the EXPLORER-HCM trial. At the same time, the longer-term efficacy of mavacamten in reducing the need for septal reduction therapy (SRT) in patients with obstructive hypertrophic cardiomyopathy (HCM) is not known.
Desai et al in a recent extended follow-up study of VALOR-HCM trial have now shown that for patients with symptomatic obstructive HCM, there is sufficient and sustained improvement with mavacamten at 56 weeks of follow-up, thereby reducing the need for SRT and representing a useful therapeutic option for patients.
To examine the cumulative longer-term effect of mavacamten on the need for SRT through week 56, the authors recruited patients from 19 US HCM centers. Included in the trial were patients with obstructive HCM (NYHA class III/IV) referred for SRT.
Patients initially assigned to mavacamten at baseline continued the drug for 56 weeks, and patients taking placebo crossed over to mavacamten from week 16 to week 56 (40-week exposure). Dose titrations were performed using echocardiographic LVOT gradient and LV ejection fraction (LVEF) measurements.
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