The U.S. Food and Drug Administration (FDA) has approved Myqorzo (aficamten) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
oHCM is a rare genetic heart condition characterized by excessive growth of heart muscle cells. The condition involves significant obstruction in the left ventricular outflow tract, leading to symptoms like dyspnea, fatigue, and potentially life-threatening cardiac events. Patients may also experience left ventricular hypertrophy, microvascular ischemia, myocardial fibrosis, and diastolic dysfunction. oHCM is often associated with genetic mutations in the MYH7 and MYBPC3 genes.
Aficamten is an allosteric cardiac myosin inhibitor approved to improve functional capacity and symptoms in patients with oHCM. Myosin inhibition has been shown to reduce cardiac contractility and LVOT obstruction.
The approval is largely based on positive results from the phase 3 SEQUIA-HCM clinical trial, recently published in the New England Journal of Medicine. In that study, treatment with aficamten for 24 weeks resulted in significant improvement in exercise capacity compared to placebo, increasing peak oxygen uptake measured by cardiopulmonary exercise testing by 1.8 mL/kg/min in the aficamten group versus 0.0 mL/kg.min in the placebo group. This effect was consistent across all prespecified subgroups.
Additionally, aficamten was well-tolerated, with no worsening heart failure or treatment interruptions due to low left ventricular ejection fraction (LVEF). Treatment emergent serious events occurred in 5.6% of patients in aficamten and 9.3% of patients on placebo.
The full U.S. Prescribing Information for aficamten includes a Boxed WARNING for the risk of heart failure. Aficamten reduces LVEF and can cause heart failure due to systolic dysfunction.
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To learn more about oHCM and other rare heart conditions, visit https://checkrare.com/diseases/heart-diseases/