Studies

VALOR-HCM: A phase 3 trial evaluating the efficacy and safety of CAMZYOS^® in SRT-eligible patients

A multicenter, randomized, double-blind, placebo-controlled trial evaluating CAMZYOS in patients with severely symptomatic obstructive HCM who were guideline eligible for SRT^1,10

Study design with dose titration regimen

Key endpoints: Primary composite and secondary endpoints^1,10

CAMZYOS^® significantly reduced the proportion of patients who proceeded with or remained guideline eligible for SRT vs placebo^1,10

CAMZYOS^® significantly improved post-exercise LVOT gradient vs placebo^1,10

Significantly more patients showed an improvement in symptom control (≥1 NYHA class) with CAMZYOS^® vs placebo^1,10

The safety profile for CAMZYOS^® was similar for EXPLORER-HCM and VALOR-HCM, with no patients permanently discontinuing treatment due to LVEF <50%¹

Most commonly reported adverse reactions with CAMZYOS^®

1.     CAMZYOS^®, Product Information current edition
2.     Olivotto I, Oreziak A, Barriales-Villa R, et al. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORERHCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2020;396(10253):759-769.
3.     Ho CY, Day SM, Ashley EA, et al. Genotype and lifetime burden of disease in hypertrophic cardiomyopathy: insights from the Sarcomeric Human Cardiomyopathy Registry (SHaRe). Circulation. 2018;138(14):1387-1398.
4.     Olivotto I, Cecchi F, Poggesi C, Yacoub MH. Patterns of disease progression in hypertrophic cardiomyopathy: an individualized approach to clinical staging. Circ Heart Fail. 2012;5(4):535-546.
5.     Argulian E, Sherrid MV, Messerli FH. Misconceptions and facts about hypertrophic cardiomyopathy. Am J Med. 2016;129(2):148-152.
6.     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;35(39):2733-2779.
7.     Marian AJ, Braunwald E. Hypertrophic cardiomyopathy: genetics, pathogenesis, clinical manifestations, diagnosis, and therapy. Circ Res. 2017;121(7):749-770.
8.     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. Circulation. 2020;142(25):e558-e631.
9.     Maron BJ, Desai MY, Nishimura RA, et al. Management of hypertrophic cardiomyopathy: JACC State-of-the-Art Review. J Am Coll Cardiol. 2022;79(4):390-414.
10.  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;80(2):95-108.
11.  Maron BJ, Olivotto I, Bellone P, et al. Clinical profile of stroke in 900 patients with hypertrophic cardiomyopathy. J Am Coll Cardiol. 2002;39(2):301-307.
12.  Maron BJ, Maron MS. Hypertrophic cardiomyopathy. Lancet. 2013;381(9862):242-255.
13.  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;239:80-89.
14.  Scott SA, Sangkuhl K, Gardner EE, et al. Clinical Pharmacogenetics Implementation Consortium guidelines for cytochrome P450-2C19 (CYP2C19) genotype and clopidogrel therapy. Clin Pharmacol Ther. 2011;90(2):328-332.