Aficamten

Last updated

Aficamten
Aficamten.svg
Clinical data
Trade names Myqorzo
Other namesCK-3773274
License data
Routes of
administration 		By mouth
Drug class Cardiac myosin inhibitor
ATC code
  • None
Legal status
Legal status
Identifiers
  • N-[(1R)-5-(5-Ethyl-1,2,4-oxadiazol-3-yl)-2,3-dihydro-1H-inden-1-yl]-1-methylpyrazole-4-carboxamide
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
PDB ligand
Chemical and physical data
Formula C18H19N5O2
Molar mass 337.383 g·mol−1
3D model (JSmol)
  • CCC1=NC(=NO1)C2=CC3=C(C=C2)[C@@H](CC3)NC(=O)C4=CN(N=C4)C
  • InChI=1S/C18H19N5O2/c1-3-16-21-17(22-25-16)12-4-6-14-11(8-12)5-7-15(14)20-18(24)13-9-19-23(2)10-13/h4,6,8-10,15H,3,5,7H2,1-2H3,(H,20,24)/t15-/m1/s1
  • Key:IOVAZWDIRCRMTM-OAHLLOKOSA-N

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]

Contents

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]

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] [7] 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]

Names

Aficamten is the international nonproprietary name. [8]

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

References

  1. 1 2 3 4 5 https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219083s000lbl.pdf
  2. ↑ Chuang, Chihyuan; Collibee, Scott; Ashcraft, Luke; Wang, Wenyue; Vander Wal, Mark; Wang, Xiaolin; 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. S2CID   238355647.
  3. ↑ Zhao, Xue; Liu, Hongzhong; Tian, Wei; Fang, Ligang; Yu, Mengyang; Wu, Xiaofei; 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 1227470. doi: 10.3389/fphar.2023.1227470 . PMC   10482267 . 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. S2CID   260348901.
  5. 1 2 3 4 "Myqorzo EPAR". European Medicines Agency (EMA). 12 December 2025. Retrieved 22 December 2025. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  6. 1 2 3 4 5 6 7 8 9 "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.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  7. ↑ "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.
  8. ↑ World Health Organization (2021). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 86". WHO Drug Information. 35 (3). hdl: 10665/346562 .

Further reading


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