FDA Approves Aficamten for Obstructive Hypertrophic Cardiomyopathy

The FDA has approved aficamten (Myqorzo; Cytokinetics), a first-in-class cardiac myosin inhibitor, for adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).¹

According to a Cytokinetics news release, this marks a significant expansion of pharmacologic options for a condition long managed with beta-blockers, calcium channel blockers, or invasive procedures.

Aficamten is an oral, selective, reversible inhibitor of cardiac myosin designed to reduce hypercontractility and left ventricular outflow tract (LVOT) obstruction—hallmark features of oHCM. The FDA previously extended the review period to allow for additional evaluation of the drug’s Risk Evaluation and Mitigation Strategy (REMS), required due to the therapy’s potential to reduce left ventricular ejection fraction (LVEF) and precipitate systolic dysfunction.

“Living with symptomatic obstructive HCM means managing physical limitations and burdensome symptoms every day of your life,” Lisa Salberg, founder and CEO of the Hypertrophic Cardiomyopathy Association (HCMA), said in the news release. “For far too long, we’ve had few options to address our needs, and the approval of MYQORZO is a long-awaited and major addition to bring new hope to patients living with oHCM.”

SEQUOIA-HCM and MAPLE-HCM Evidence of Aficamten

This FDA approval was supported by data from the placebo-controlled SEQUOIA-HCM trial (NCT05186818) and the active-comparator MAPLE-HCM study (NCT05767346), which together evaluated aficamten in both more symptomatic and less-severe patient populations.^2,3

In SEQUOIA-HCM, aficamten met its primary end point at 24 weeks, significantly improving peak oxygen uptake (pVO₂) by 1.8 ml per kilogram on average compared with no increase with placebo.² Patients receiving aficamten also achieved meaningful gains in New York Heart Association functional class and Kansas City Cardiomyopathy Questionnaire clinical summary scores, reflecting improvements in daily symptoms and health status. Safety outcomes were generally comparable between groups, with low rates of treatment-emergent serious adverse events.

MAPLE-HCM extended these findings by comparing aficamten head-to-head with metoprolol, a long-standing first-line therapy for oHCM.³ At 24 weeks, aficamten was associated with superior improvements in exercise capacity and multiple secondary end points, including LVOT gradients and cardiac biomarkers, without an excess of adverse events. Importantly, the trial enrolled patients with less severe disease, supporting the drug’s efficacy across a wider clinical spectrum.

Labeling, Availability, and Clinical Considerations of Aficamten

The approved prescribing information for aficamten includes a boxed warning for heart failure due to systolic dysfunction.¹ Echocardiographic monitoring is required before and during therapy, and treatment initiation is not recommended in patients with LVEF below 55%. Dose reductions or interruptions are mandated if LVEF declines or if heart failure symptoms emerge. As a result, aficamten will be dispensed only through a restricted REMS program. Despite these safeguards, Cytokinetics emphasized the drug’s flexible dosing and lack of required drug-drug interaction monitoring as practical advantages for clinicians.

Aficamten is expected to become available in the US in the second half of January 2026, with patient support offered through the company’s Myqorzo & You program.

References

1. Cytokinetics announces FDA approval of Myqorzo™ (aficamten) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy to improve functional capacity and symptoms. News release. Cytokinetics. December 19, 2025. Accessed December 22, 2025. https://ir.cytokinetics.com/press-releases/press-release-details/2025/Cytokinetics-Announces-FDA-Approval-of-MYQORZO-aficamten-for-the-Treatment-of-Adults-with-Symptomatic-Obstructive-Hypertrophic-Cardiomyopathy-to-Improve-Functional-Capacity-and-Symptoms/default.aspx
2. Maron MS, Masri A, Nassif ME, et al. Aficamten for symptomatic obstructive hypertrophic cardiomyopathy. N Engl J Med. 2024;390(20):1849-1861. doi:10.1056/NEJMoa2401424
3. Garcia-Pavia P, Maron MS, Masri A, et al. Aficamten or metoprolol monotherapy for obstructive hypertrophic cardiomyopathy. N Engl J Med. 2025;393(10):949-960. doi:10.1056/NEJMoa2504654