Action

The U.S. Food and Drug Administration (FDA) approved Myqorzo (aficamten) tablets to treat adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms.

Disease or Condition

Symptomatic oHCM is an inherited condition where patients have thickened heart muscle and reduced blood flow from the left side of the heart to the rest of the body, causing symptoms such as shortness of breath, fatigue, and potentially life-threatening cardiac events.

Myqorzo Effectiveness

The effectiveness and safety of Myqorzo were studied in 282 adults with symptomatic oHCM randomly assigned to receive Myqorzo or placebo for 24 weeks. At the end of the study, participants receiving Myqorzo had an increase in exercise capacity measured by peak oxygen uptake compared to no change in exercise capacity among those receiving placebo. Also, 59 percent of participants receiving Myqorzo experienced an improvement in physical activity limitations (measured using the New York Heart Association Classification system) compared to 24 percent of individuals receiving placebo.

Safety Information

Myqorzo reduces contraction of the heart muscle which can cause heart failure. Because of the risk for heart failure, patients treated with Myqorzo must be monitored with echocardiograms, an imaging test that shows how well the heart is working. Because of the heart failure risk and need for monitoring, Myqorzo is only available through a restricted program called the Myqorzo Risk Evaluation and Mitigation Strategy (REMS).

Patients must also avoid certain prescription medicines that interfere with the metabolism (breakdown) of Myqorzo.

See the prescribing information for additional information on risks associated with Myqorzo.

Designations

Myqorzo received Orphan Drug Designation and Breakthrough Therapy Designation for this indication.