Synonyms: Camzyos® | MYK-461 | MYK461
mavacamten is an approved drug (FDA (2022), EMA (2023))
Compound class:
Synthetic organic
Comment: Mavacamten (MYK-461) is an orally active myosin allosteric modulator [1] that was developed by MyoKardia as a novel treatment for obstructive hypertrophic cardiomyopathy (HCM). In 2020 MyoKardia was bought by Bristol Myers Squibb. The compound interacts with both α and β cardiac myosins in vitro. Preclinical pharmacokinetics are reported in [2]. Mavacamten attenuates hypertrophic and profibrotic gene expression in rodent models of HCM [1].
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References |
1. Green EM, Wakimoto H, Anderson RL, Evanchik MJ, Gorham JM, Harrison BC, Henze M, Kawas R, Oslob JD, Rodriguez HM et al.. (2016)
A small-molecule inhibitor of sarcomere contractility suppresses hypertrophic cardiomyopathy in mice. Science, 351 (6273): 617-21. [PMID:26912705] |
2. Grillo MP, Erve JCL, Dick R, Driscoll JP, Haste N, Markova S, Brun P, Carlson TJ, Evanchik M. (2019)
In vitro and in vivo pharmacokinetic characterization of mavacamten, a first-in-class small molecule allosteric modulator of beta cardiac myosin. Xenobiotica, 49 (6): 718-733. [PMID:30044681] |
3. Heitner SB, Jacoby D, Lester SJ, Owens A, Wang A, Zhang D, Lambing J, Lee J, Semigran M, Sehnert AJ. (2019)
Mavacamten Treatment for Obstructive Hypertrophic Cardiomyopathy: A Clinical Trial. Ann Intern Med, 170 (11): 741-748. [PMID:31035291] |
4. Olivotto I, Oreziak A, Barriales-Villa R, Abraham TP, Masri A, Garcia-Pavia P, Saberi S, Lakdawala NK, Wheeler MT, Owens A et al.. (2020)
Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet, 396 (10253): 759-769. [PMID:32871100] |