Chemistry:Aficamten

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Aficamten, sold under the brand name Myqorzo, is a medication used for the treatment of symptomatic obstructive hypertrophic cardiomyopathy.[1] It is a cardiac myosin inhibitor[2] developed by Cytokinetics.[3][4]

Aficamten binds directly to the motor domain of cardiac myosin and prevents it from entering the force-producing state.[5] This lowers cardiac contractility, leading to reduced left ventricular outflow tract obstruction in people with hypertrophic cardiomyopathy.[5]

Aficamten was approved for medical use in the United States in December 2025,[6] and in the European Union in February 2026.[5][7]

Medical uses

Aficamten is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy to improve functional capacity and symptoms.[1][6]

Symptomatic obstructive hypertrophic cardiomyopathy is an inherited condition where people have thickened heart muscle and reduced blood flow from the left side of the heart to the rest of the body, causing symptoms such as shortness of breath, fatigue, and potentially life-threatening cardiac events.[6]

Contraindiations

Use with rifampin is contraindicated.[1]

Adverse effects

The US prescription label for aficamten contains a boxed warning that it reduces left ventricular ejection fraction and can cause heart failure due to systolic dysfunction.[1]

History

The effectiveness and safety of aficamten were studied in 282 adults with symptomatic obstructive hypertrophic cardiomyopathy randomly assigned to receive aficamten or placebo for 24 weeks.[6] At the end of the study, participants receiving aficamten had an increase in exercise capacity measured by peak oxygen uptake compared to no change in exercise capacity among those receiving placebo.[6] Also, 59 percent of participants receiving aficamten experienced an improvement in physical activity limitations (measured using the New York Heart Association Classification system) compared to 24 percent of individuals receiving placebo.[6]

Society and culture

Aficamten was approved for medical use in the United States in December 2025.[6][8] The US Food and Drug Administration granted the application for aficamten orphan drug and breakthrough therapy designations.[6]

In December 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Myqorzo, intended for the treatment of adults with obstructive hypertrophic cardiomyopathy.[5] The applicant for this medicinal product is Cytokinetics (Ireland) Limited.[5] Aficamten was authorized for medical use in the European Union in February 2026.[5][7]

Names

Aficamten is the international nonproprietary name.[9]

Aficamten is sold under the brand name Myqorzo.[6]

References

  1. ↑ 1.0 1.1 1.2 1.3 Cite error: Invalid <ref> tag; no text was provided for refs named Myqorzo FDA label
  2. ↑ Chuang, Chihyuan; Collibee, Scott; Ashcraft, Luke; Wang, Wenyue; Vander Wal, Mark; Wang, Xiaolin; Hwee, Darren T.; Wu, Yangsong et al. (October 2021). "Discovery of Aficamten (CK-274), a Next-Generation Cardiac Myosin Inhibitor for the Treatment of Hypertrophic Cardiomyopathy". Journal of Medicinal Chemistry 64 (19): 14142–14152. doi:10.1021/acs.jmedchem.1c01290. ISSN 0022-2623. PMID 34606259. 
  3. ↑ Zhao, Xue; Liu, Hongzhong; Tian, Wei; Fang, Ligang; Yu, Mengyang; Wu, Xiaofei; Liu, Aijing; Wan, Ruijie et al. (2023). "Safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of aficamten in healthy Chinese participants: a randomized, double-blind, placebo-controlled, phase 1 study". Frontiers in Pharmacology 14. doi:10.3389/fphar.2023.1227470. PMID 37680714. 
  4. ↑ Sebastian, Sneha Annie; Padda, Inderbir; Lehr, Eric J.; Johal, Gurpreet (September 2023). "Aficamten: A Breakthrough Therapy for Symptomatic Obstructive Hypertrophic Cardiomyopathy". American Journal of Cardiovascular Drugs: Drugs, Devices, and Other Interventions 23 (5): 519–532. doi:10.1007/s40256-023-00599-0. ISSN 1179-187X. PMID 37526885. 
  5. ↑ 5.0 5.1 5.2 5.3 5.4 5.5 "Myqorzo EPAR". 12 December 2025. https://www.ema.europa.eu/en/medicines/human/EPAR/myqorzo.  Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  6. ↑ 6.0 6.1 6.2 6.3 6.4 6.5 6.6 6.7 6.8 "FDA approves drug to improve functional capacity and symptoms in adults with rare inherited heart condition". U.S. Food and Drug Administration (FDA) (Press release). 22 December 2025. Retrieved 22 December 2025. Public Domain This article incorporates text from this source, which is in the public domain.
  7. ↑ 7.0 7.1 "Myqorzo Product information". 13 February 2026. https://ec.europa.eu/health/documents/community-register/html/h2014.htm. 
  8. ↑ "Cytokinetics Announces FDA Approval of Myqorzo (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy to Improve Functional Capacity and Symptoms" (Press release). Cytokinetics. 19 December 2025. Retrieved 22 December 2025 – via GlobeNewswire News Room.
  9. ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 86". WHO Drug Information 35 (3). 2021. 

Further reading

  • Maron, Martin S.; Masri, Ahmad; Choudhury, Lubna; Olivotto, Iacopo; Saberi, Sara; Wang, Andrew; Garcia-Pavia, Pablo; Lakdawala, Neal K. et al. (January 2023). "Phase 2 Study of Aficamten in Patients With Obstructive Hypertrophic Cardiomyopathy". Journal of the American College of Cardiology 81 (1): 34–45. doi:10.1016/j.jacc.2022.10.020. PMID 36599608. 
  • Clinical trial number NCT05186818 for "Aficamten vs Placebo in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy (SEQUOIA-HCM) (SEQUOIA-HCM)" at ClinicalTrials.gov



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