Patent No. EP3661514 (titled "Mavacamten For Use In The Treatment Of Hypertrophic Cardiomyopathy") was filed by Myokardia on Aug 3, 2018. The application was issued on Sep 17, 2025.
Compound 1, or a pharmaceutically acceptable salt thereof, is used to treat hypertrophic cardiomyopathy (HCM) and myocardial diastolic dysfunction. The method comprises administering a therapeutically effective amount, specifically a total daily dosage of 2 mg to 15 mg ± 10%, including specific doses of 2.5 mg, 5 mg, 10 mg, or 15 mg. Treatment may target obstructive HCM (oHCM) with LVOT gradients ≥30 mmHg or non-obstructive HCM and HFpEF. Efficacy is associated with maintaining blood plasma concentrations typically between 200 and 750 ng/mL (optimally 350–700 ng/mL). Dosage may be adjusted based on plasma levels, subject weight (e.g., 10 mg for ≤60 kg; 15 mg for >60 kg), or changes in left ventricular ejection fraction (LVEF). Compound 1 may be administered concomitantly with beta-blockers.

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