FDA Approves Aficamten for Obstructive HCM

The US Food and Drug Administration has approved aficamten (Myqorzo) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM), drug manufacturer Cytokinetics announced last week.

The allosteric and reversible inhibitor of cardiac myosin motor activity improved exercise capacity along with symptoms and quality of life, while also being well tolerated, in the SEQUOIA-HCM trial. Nearly 60% of those randomized to aficamten had an improvement in physical activity limitations compared with 24% of those randomized to placebo.

In announcing the FDA approval, Cytokinetics said it represents “an important step forward in how we care for people living with obstructive HCM.”

Aficamten is the second cardiac myosin inhibitor to get regulatory signoff in the United States, following the approval in April 2022 of mavacamten (Camzyos, Bristol Myers Squibb). The approval is the first ever for Cytokinetics.

Prior to the availability of these new drugs, options were slim and included medical management with beta-blockers or calcium channel blockers, as well as disopyramide (Norpace; Pfizer), to control symptoms. Failing that, invasive options included alcohol septal ablation or surgical myectomy.

Like mavacamten, aficamten is only available through the FDA’s Risk Evaluation and Mitigation Strategy (REMS) program and contains a warning to avoid certain prescription drugs that could interfere with the metabolism of cardiac myosin inhibitors.