FDA Approves First-in-Class Therapy, Myqorzo, for Obstructive Hypertrophic Cardiomyopathy
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A new treatment option is now available for adults battling symptomatic obstructive hypertrophic cardiomyopathy (oHCM), offering hope for improved functional capacity and symptom relief. The Food and Drug Administration approved aficamten (Myqorzo; Cytokinetics) on December 19, 2025, marking a significant advancement in the management of this challenging heart condition.
A Long-Awaited Breakthrough for oHCM Patients
For years, individuals with oHCM have primarily relied on medications like beta-blockers and calcium channel blockers, or faced the prospect of invasive procedures to manage their symptoms. “Living with symptomatic obstructive HCM means managing physical limitations and burdensome symptoms every day of your life,” stated Lisa Salberg, founder and CEO of the Hypertrophic Cardiomyopathy Association (HCMA), in a company 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.”
How Aficamten Works
Aficamten is an oral medication classified as a cardiac myosin inhibitor. It selectively and reversibly inhibits cardiac myosin, a protein crucial for heart muscle contraction. By reducing hypercontractility and alleviating left ventricular outflow tract (LVOT) obstruction – key characteristics of oHCM – aficamten aims to improve heart function and reduce symptoms.The FDA’s review process was extended to thoroughly evaluate the drug’s Risk Evaluation and Mitigation Strategy (REMS), necessitated by its potential to lower left ventricular ejection fraction (LVEF) and potentially lead to systolic dysfunction.
Clinical Trial Evidence: SEQUOIA-HCM and MAPLE-HCM
The FDA’s decision was based on robust data from two pivotal clinical trials: the placebo-controlled SEQUOIA-HCM trial (NCT05186818) and the active-comparator MAPLE-HCM study (NCT05767346). these trials assessed aficamten’s efficacy in diverse patient populations, ranging from those with more severe symptoms to individuals with less-severe disease.
In SEQUOIA-HCM, participants receiving aficamten demonstrated a statistically significant enhancement in peak oxygen uptake (pVO) by 1.8 ml per kilogram on average at 24 weeks, compared to no change in the placebo group.moreover, patients experienced improvements in New York Heart Association (NYHA) functional class and Kansas City Cardiomyopathy Questionnaire (KCCQ) scores. The MAPLE-HCM trial, published in 2025, compared aficamten to metoprolol and demonstrated superior efficacy in improving pVO and KCCQ scores.
Supporting Research
- Cytokinetics Announces FDA Approval of MYQORZO (aficamten)
- 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
- 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
This approval represents a significant step forward in the treatment of oHCM, offering a new therapeutic avenue for patients seeking to improve their quality of life.
