Advertisement

Efficacy and safety of Mavacamten: A new era in the treatment of hypertrophic cardiomyopathy

Published:August 29, 2022DOI:https://doi.org/10.1016/j.ejim.2022.08.023
      Hypertrophic cardiomyopathy (HCM) is a condition in which the myocardium is hypertrophied. HCM is the most common genetic cardiovascular disease and the most common cause of death in young adults [
      • Basit H.
      • Brito D.
      • Sharma S
      Hypertrophic cardiomyopathy.
      ]. The heart muscle thickens mostly in the septum and obstructs blood flow from the heart to the body Patients at risk of HCM, can be treated with prophylactic implantation of an implantable cardiac defibrillator (ICD). In addition, the treatment options for HOCM include beta-blocker or calcium channel blockers such as verapamil [
      • Prinz C.
      • Farr M.
      • Hering D.
      • Horstkotte D.
      • Faber L.
      The diagnosis and treatment of hypertrophic cardiomyopathy.
      ]. Failure of conservative management requires invasive procedures such as surgical myectomy and percutaneous septal ablation [
      • Prinz C.
      • Farr M.
      • Hering D.
      • Horstkotte D.
      • Faber L.
      The diagnosis and treatment of hypertrophic cardiomyopathy.
      ]. Current studies show that FDA-approved Mavacamten, an orally administered cardiac myosin inhibitor, is effective in patients with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy [

      FDA approves new drug to improve heart function in adults with rare heart condition [Internet]. U.S. Food and Drug Administration. FDA; [cited 2022 Jul 29]. Available from: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-drug-improve-heart-function-adults-rare-heart-condition.

      ].
      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'

      Subscribe:

      Subscribe to European Journal of Internal Medicine
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Basit H.
        • Brito D.
        • Sharma S
        Hypertrophic cardiomyopathy.
        StatPearls. StatPearls Publishing, 2022 ([Internet])
        • Prinz C.
        • Farr M.
        • Hering D.
        • Horstkotte D.
        • Faber L.
        The diagnosis and treatment of hypertrophic cardiomyopathy.
        Dtsch Arztebl Int. 2011; 108: 209-215https://doi.org/10.3238/arztebl.2011.0209
      1. FDA approves new drug to improve heart function in adults with rare heart condition [Internet]. U.S. Food and Drug Administration. FDA; [cited 2022 Jul 29]. Available from: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-drug-improve-heart-function-adults-rare-heart-condition.

        • Olivotto I.
        • Oreziak A.
        • Barriales-Villa R.
        • Abraham T.P.
        • Masri A.
        • Garcia-Pavia P.
        • et al.
        Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial.
        Lancet. 2020; 396: 759-769https://doi.org/10.1016/S0140-6736(20)31792-X
        • Ho C.Y.
        • Mealiffe M.E.
        • Bach R.G.
        • Bhattacharya M.
        • Choudhury L.
        • Edelberg J.M.
        • et al.
        Evaluation of mavacamten in symptomatic patients with nonobstructive hypertrophic cardiomyopathy.
        J Am Coll Cardiol. 2020; 75: 2649-2660https://doi.org/10.1016/j.jacc.2020.03.064
      2. Officer C.E. RoB 2: a revised Cochrane risk-of-bias tool for randomized trials [Internet]. Cochrane.org [cited 2022 Jul 29]. Available from: https://methods.cochrane.org/bias/resources/rob-2-revised-cochrane-risk-bias-tool-randomized-trials.

        • Hegde S.M.
        • Lester S.J.
        • Solomon S.D.
        • Michels M.
        • Elliott P.M.
        • Nagueh S.F.
        • et al.
        Effect of mavacamten on echocardiographic features in symptomatic patients with obstructive hypertrophic cardiomyopathy.
        J Am Coll Cardiol. 2021; 78: 2518-2532https://doi.org/10.1016/j.jacc.2021.09.1381
        • Desai M.Y.
        • Wolski K.
        • Owens A.
        • Naidu S.S.
        • Geske J.B.
        • Smedira N.G.
        • et al.
        Study design and rationale of VALOR-HCM: evaluation of mavacamten in adults with symptomatic obstructive hypertrophic cardiomyopathy who are eligible for septal reduction therapy.
        Am Heart J. 2021; 239: 80-89https://doi.org/10.1016/j.ahj.2021.05.007
        • Heitner S.B.
        • Jacoby D.
        • Lester S.J.
        • Owens A.
        • Wang A.
        • Zhang D.
        • et al.
        Mavacamten treatment for obstructive hypertrophic cardiomyopathy: a clinical trial: a clinical trial.
        Ann Intern Med. 2019; 170: 741-748https://doi.org/10.7326/M18-3016
        • Kawas R.F.
        • Anderson R.L.
        • Ingle S.R.B.
        • Song Y.
        • Sran A.S.
        • Rodriguez H.M.
        A small-molecule modulator of cardiac myosin acts on multiple stages of the myosin chemomechanical cycle.
        J Biol Chem. 2017; 292: 16571-16577https://doi.org/10.1074/jbc.m117.776815