An evaluation of mavacamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy in adults

References

• Heitner SB, Jacoby D, Lester SJ, et al. Mavacamten treatment for obstructive hypertrophic cardiomyopathy. Ann Intern Med. 2019;170(11):741–748.
   PubMed Web of Science ®Google Scholar
• Maron BJ, Longo DL. Clinical course and management of hypertrophic cardiomyopathy. N Engl J Med. 2018;379(7):655–668.
   PubMed Web of Science ®Google Scholar
• Reant P, Dufour M, Peyrou J, et al. Upright treadmill vs. semi-supine bicycle exercise echocardiography to provoke obstruction in symptomatic hypertrophic cardiomyopathy: a pilot study. Eur Heart J Cardiovasc Imaging. 2018 Jan 1;19(1):31–38.
   PubMed Web of Science ®Google Scholar
• Maron MS, Olivotto I, Zenovich AG, et al. Hypertrophic cardiomyopathy is predominantly a disease of left ventricular outflow tract obstruction. Circulation. 2006 Nov 21;114(21):2232–2239.
   PubMed Web of Science ®Google Scholar
• Dimitrow PP, Cheng TO. Standing position alone or in combination with exercise as a stress test to provoke left ventricular outflow tract gradient in hypertrophic cardiomyopathy and other conditions. Int J Cardiol. 2010 Sep 3;143(3):219–222.
   PubMed Web of Science ®Google Scholar
• Kim LK, Swaminathan RV, Looser P, et al. Hospital volume outcomes after septal myectomy and alcohol septal ablation for treatment of obstructive hypertrophic cardiomyopathy: US nationwide inpatient database, 2003-2011. JAMA Cardiol. 2016 Jun 1;1(3):324–332.
   PubMed Web of Science ®Google Scholar
• Ommen SR, Mital S, Burke MA, et al. 2020 AHA/ACC guideline for the diagnosis and treatment of patients with hypertrophic cardiomyopathy: a report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines. J Am Coll Cardiol. 2020;76(25):e159–e240.
   PubMed Web of Science ®Google Scholar
• Epstein SE, Rosing DR. Verapamil: its potential for causing serious complications in patients with hypertrophic cardiomyopathy. Circulation. 1981 Sep;64(3):437–441.
   PubMed Web of Science ®Google Scholar
• Sherrid MV. Drug therapy for hypertrophic cardiomyopathy: physiology and practice. Curr Cardiol Rev. 2016;12(1):52–65.
   PubMedGoogle Scholar
• Elliott PM, Anastasakis A, Borger MA, et al. 2014 ESC guidelines on diagnosis and management of hypertrophic cardiomyopathy: the task force for the diagnosis and management of hypertrophic cardiomyopathy of the European Society of Cardiology (ESC). Eur Heart J. 2014 Oct 14;35(39):2733–2779.
   PubMed Web of Science ®Google Scholar
• Edelberg JM, Sehnert AJ, Mealiffe ME, et al. The impact of mavacamten on the pathophysiology of hypertrophic cardiomyopathy: a narrative review. Am J Cardiovasc Drugs. 2022 Sep;22(5):497–510.
   PubMed Web of Science ®Google Scholar
• Grillo MP, Erve JCL, Dick R, et al. In vitro and in vivo pharmacokinetic characterization of mavacamten, a first-in-class small molecule allosteric modulator of beta cardiac myosin. Xenobiotica. 2019 Jun;49(6):718–733.
   PubMed Web of Science ®Google Scholar
• Kawas RF, Anderson RL, Ingle SRB, et al. A small-molecule modulator of cardiac myosin acts on multiple stages of the myosin chemomechanical cycle. J Biol Chem. 2017 Oct 6;292(40):16571–16577.
   PubMed Web of Science ®Google Scholar
• Administration FD. Approved drug proucts: CAMZYOS (mavacamten) capsules for oral use [Webpage]. 2022. [cited 2022 Oct 15]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/214998s000lbl.pdf.
   Google Scholar
• Green EM, Wakimoto H, Anderson RL, et al. A small-molecule inhibitor of sarcomere contractility suppresses hypertrophic cardiomyopathy in mice. Science (New York, NY). 2016 Feb 5;351(6273):617–621.
   Google Scholar
• Mamidi R, Li J, Doh CY, et al. Impact of the myosin modulator mavacamten on force generation and cross-bridge behavior in a murine model of hypercontractility. J Am Heart Assoc. 2018 Sep 4;7(17):e009627.
   PubMed Web of Science ®Google Scholar
• Stern JA, Markova S, Ueda Y, et al. A small molecule inhibitor of sarcomere contractility acutely relieves left ventricular outflow tract obstruction in feline hypertrophic cardiomyopathy. PloS One. 2016;11(12):e0168407.
   PubMed Web of Science ®Google Scholar
• Olivotto I, Oreziak A, Barriales-Villa R, et al. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2020;396(10253):759–769.
   PubMed Web of Science ®Google Scholar
• Spertus JA, Fine JT, Elliott P, et al. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): health status analysis of a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2021;397(10293):2467–2475.
   PubMed Web of Science ®Google Scholar
• Rader F, Choudhury L, Saberi S, et al. Long-term safety of mavacamten in patients with obstructive hypertrophic cardiomyopathy: interim results of the mava-Long Term Extension (LTE) study. J Am Coll Cardiol. 2021;77(18_Supplement_1):532.
   Google Scholar
• Rajtar-Salwa R, Hładij R, Dimitrow PP. Elevated level of troponin but Not N-terminal probrain natriuretic peptide is associated with increased risk of sudden cardiac death in hypertrophic cardiomyopathy calculated according to the ESC guidelines 2014. Dis Markers. 2017;2017:9417908.
   PubMed Web of Science ®Google Scholar
• Desai MY, Owens A, Geske JB, et al. Myosin inhibition in patients with obstructive hypertrophic cardiomyopathy referred for septal reduction therapy. J Am Coll Cardiol. 2022 Jul 12;80(2):95–108.
   PubMed Web of Science ®Google Scholar
• Cremer PC, Geske JB, Owens A, et al. Myosin inhibition and left ventricular diastolic function in patients with obstructive hypertrophic cardiomyopathy referred for septal reduction therapy: insights from the VALOR-HCM study. Cir Cardiovasc Imaging. 2022;e014986. https://doi.org/10.1161/CIRCIMAGING.122.014986. •• This is a substudy of VALOR HCM showing improved diastolic parameters in patients randomized to the mavacamten group.
   Google Scholar
• Desai MY, Owens AT, Geske JB, et al. Dose-blinded myosin inhibition in patients with obstructive HCM referred for septal reduction therapy: outcomes through 32-weeks. Circulation. 2022. https://www.ahajournals.org/doi/10.1161/CIRCULATIONAHA.122.062534
   Google Scholar
• FDA approves new drug to improve heart function in adults with rare heart condition Internet]. FDA: Federal Drug Administration; 2022 [cited 2022 Oct 17]. Available from: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-drug-improve-heart-function-adults-rare-heart-condition
   Google Scholar
• Desai MY, Wolski K, Owens A, 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 Sep;239:80–89.
   PubMed Web of Science ®Google Scholar
• Dybro AM, Rasmussen TB, Nielsen RR, et al. Randomized trial of metoprolol in patients with obstructive hypertrophic cardiomyopathy. J Am Coll Cardiol. 2021;78(25):2505–2517.
   PubMed Web of Science ®Google Scholar
• PIONEER-LTE Study Methods. NCT03496168. ClinicalTrials.gov. [cited 2022 Oct 18]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT03496168?term=pioneer-ole&draw=2&rank=1.
   Google Scholar
• MAVA-LTE Study Methods. NCT03723655. ClinicalTrials. gov. [updated August 2022; cited 2022 Oct 17]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT03723655?term=MAVA-LTE&draw=2&rank=1.
   Google Scholar
• Ho CY, Mealiffe ME, Bach RG, et al. Evaluation of mavacamten in symptomatic patients with nonobstructive hypertrophic cardiomyopathy. J Am Coll Cardiol. 2020;75(21):2649–2660.
   PubMed Web of Science ®Google Scholar
• Patel RB, Shah SJ. Inclusion criteria for heart failure with preserved ejection fraction clinical trials: making the case for precision diagnosis and greater inclusivity. J Card Fail. 2022;28(5):732–735.
   PubMed Web of Science ®Google Scholar
• A study of mavacamten in participants with HFpEF and elevation of NT-proBNP with or without elevation of cTnT (EMBARK-HFpEF). NCT04766892. Clinicaltrials.gov [cited 2022 Oct 15]. Available from: https://clinicaltrials.gov/ct2/show/NCT04766892.
   Google Scholar
• Beinfeld M, Wasfy JH, Walton S, et al. Mavacamten for hypertrophic cardiomyopathy: effectiveness and value. J Manag Care Spec Pharm. 2022 Mar;28(3):369–375.
   PubMed Web of Science ®Google Scholar
• REDWOOD-HCM Study Methods. NCT04219826. Clinical Trials.gov. [cited 2022 Oct 15]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT04219826?term=REDWOOD-HCM&draw=2&rank=1.
   Google Scholar