FDA Approves New Treatment for Rare Heart Condition

The US Food and Drug Administration (FDA) has approved a new medication therapy, Camzyos (mavacamten), to treat symptomatic individuals with class II-III obstructive hypertrophic cardiomyopathy (oHCM).

The approval was based on a study in which adults with oHCM were randomized to receive mavacamten in 2.5, 5, 10, or 15 mg capsules or placebo daily for 30 weeks. The results of the study indicated that 37% of the mavacamten group had improved exercise capacity and symptoms at the end of the study period, vs 17% of the placebo group.

Individuals with a serious intercurrent illness or arrhythmia who take mavacamten may be at an increased risk of developing impaired heart muscle contraction, heart failure, and blocked functioning of the ventricles. As a result of the risk of heart failure, mavacamten is only available for use through the Camzyos Risk Evaluation and Mitigation Strategy (REMS) restricted program.

All patients treated with mavacamten should be monitored with echocardiograms. Patients should also avoid certain prescription and over-the-counter medicines while using mavacamten.

This indication has received breakthrough therapy and orphan drug designations.


—Leigh Precopio



FDA approves new drug to improve heart function in adults with rare heart condition. News release. US Food and Drug Administration; April 29, 2022. Accessed May 10, 2022.