The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for Myqorzo (aficamten, Cytokinetics) for treating adults with obstructive hypertrophic cardiomyopathy (oHCM). 

Hypertrophic cardiomyopathy is the most common form of genetic cardiovascular disease and the most common cause of sudden death in young people. It is caused by mutations in genes encoding the cardiac sarcomere protein. oHCM happens 

when thickened heart muscle and abnormal motion of the mitral valve block blood flow from the left ventricle.

A recent analysis of six European countries found that 0.027%-0.069% of the population have oHCM, with men generally more affected than women. 

The active substance in Myqorzo is aficamten, a reversible cardiac myosin inhibitor that binds to cardiac myosin’s motor domain to prevent it from entering the force-producing state. This reduces cardiac contractility, which in turn lessens left ventricular outflow tract obstruction. 

The CHMP’s recommendation comes after results from a phase 3, randomized, placebo-controlled trial involving 282 patients with symptomatic oHCM. They were given either aficamten or placebo daily for 24 weeks. 

After 24 weeks, 68% of patients treated with aficamten exhibited a complete hemodynamic response compared with 7% of those taking the placebo. Those taking aficamten were also more likely to experience improvement in limiting symptoms and enhanced exercise capacity than those taking the placebo. 

Furthermore, 88% of participants treated with aficamten compared with 52% of those taking the placebo were no longer eligible for septal reduction therapy after 24 weeks of treatment. 

The most common side effects from Myqorzo include dizziness, hypertension, palpitations, and systolic dysfunction defined as left ventricular ejection fraction below 50%. 

Myqorzo will be available as 5 mg, 10 mg, 15 mg, and 20 mg film-coated tablets. Treatment should be initiated by physicians with experience in managing patients with cardiomyopathy. 

Detailed recommendations for use of Myqorzo will be available in the summary of product characteristics, to be published on the EMA’s website after marketing authorization is granted by the European Commission.

Annie Lennon is a medical journalist. Her writing appears on Medscape, WebMD, and Medical News Today, among other outlets.