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Mavacamten in obstructive hypertrophic cardiomyopathy: a profile of its use

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Abstract

Mavacamten (Camzyos®), an oral, once-daily cardiac myosin inhibitor, is an important new option for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). In the USA, mavacamten is the first approved drug for the treatment of adults with symptomatic New York Heart Association (NYHA) class II–III obstructive HCM to improve functional capacity and symptoms. In a pivotal phase 3 trial in this patient population, mavacamten improved exercise capacity, symptom burden, and left ventricular outflow tract obstruction compared with placebo. Mavacamten was generally well tolerated, with the most common adverse events being dizziness and syncope. Longer-term data up to 84 weeks demonstrated that the efficacy of mavacamten was maintained with no new safety concerns. Mavacamten may increase the risk of heart failure due to systolic dysfunction and therefore, individualized dosage and close monitoring is required.

Plain Language Summary

Obstructive hypertrophic cardiomyopathy (HCM) is a heart disorder characterized by increased contractility, thickening of the left ventricle wall, and left ventricular outflow tract obstruction. Symptoms of obstructive HCM include chest pain and shortness of breath during physical activity. Mavacamten (Camzyos®) is the first drug specifically approved for obstructive HCM in adults, which reduces contractility by inhibiting cardiac myosin. In a clinical trial, mavacamten improved exercise capacity and symptoms in patients with obstructive HCM and New York Heart Association (NYHA) class II–III symptoms. Mavacamten was generally well tolerated. Data up to 84 weeks from the long-term extension study show that the efficacy and tolerability of mavacamten were sustained. Given that mavacamten may increase the risk of heart failure due to systolic dysfunction, it should be administered at an individualized dosage with close monitoring. Mavacamten is an important new treatment option for patients who experience symptoms due to obstructive HCM.

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Acknowledgements

The manuscript was reviewed by: Z. Ali, Pharmacy Department, Hamad Medical Corporation, Doha, Qatar; S. Chinwong, Department of Pharmaceutical Care, Chiang Mai University, Chiang Mai, Thailand; L. Choudhury, Bluhm Cardiovascular Institute, Division of Cardiology, Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA; H. Parthasarathy, Department of Cardiology, Apollo Hospitals, Chennai, India; S. J. Rao, Department of Medicine, MedStar Union Memorial Hospital, Baltimore, MD, USA. During the peer review process, Bristol-Myers Squibb Company, the marketing authorization holder of mavacamten, was also offered an opportunity to provide a scientific accuracy review of their data. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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    Tina Nie & Yahiya Y. Syed

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Correspondence to Tina Nie.

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Tina Nie and Yahiya Syed are salaried employees of Adis International Ltd/Springer Nature and declare no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

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Nie, T., Syed, Y.Y. Mavacamten in obstructive hypertrophic cardiomyopathy: a profile of its use. Drugs Ther Perspect 39, 223–231 (2023). https://doi.org/10.1007/s40267-023-01003-2

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