FDA Approves Myqorzo for Symptomatic Obstructive Hypertrophic Cardiomyopathy

MONDAY, Jan. 5, 2026 (HealthDay News) — The U.S. Food and Drug Administration has approved Myqorzo (aficamten) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

Myqorzo is a cardiac myosin inhibitor that directly addresses underlying hypercontractility associated with oHCM and aids left ventricular outflow tract obstruction. Myqorzo is available in 5-, 10-, 15-, and 20-mg tablets and the U.S. Prescribing Information for Myqorzo includes a boxed warning for the risk of heart failure. Myqorzo reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction, making echocardiogram assessments required before and during treatment to monitor for systolic dysfunction.

The approval is based on the phase 3 SEQUOIA-HCM clinical trial that showed treatment with Myqorzo for 24 weeks significantly improved exercise capacity and increased peak oxygen uptake by 1.8 mL/kg/minute compared with baseline, versus 0.0 mL/kg/minute in patients treated with placebo (least square mean difference, 1.74 mL/kg/minute). The treatment effect of Myqorzo was persistent across all prespecified subgroups (e.g., age, sex, patient baseline characteristics) and regardless of background beta-blocker therapy.

Myqorzo was well tolerated, with no instances noted of worsening heart failure or treatment interruptions due to low LVEF. Treatment-emergent serious adverse events occurred in 5.6 percent of patients treated with Myqorzo versus 9.3 percent taking placebo. Hypertension was the only adverse reaction occurring in >5 percent of patients, more commonly seen in patients on Myqorzo versus placebo (8 versus 2 percent).

“I”m pleased that the approved label and REMS [Risk Evaluation and Mitigation Strategy] reflect the distinct characteristics of Myqorzo, including a straightforward, flexible dosing regimen, no requirement for drug-drug interaction monitoring, and a predictable safety profile,” Robert I. Blum, the president and chief executive officer of Cytokinetics, said in a statement.

Approval of Myqorzo was granted to Cytokinetics.

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