Comprehensive Review: Mavacamten and Aficamten in Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy (HCM) is the most common monogenic heart disease, with an estimated prevalence of 1:600 in the general population, and is associated with significant morbidity. HCM is characterized by left ventricular hypertrophy and interventricular septal thickening due to sarcomere pr...
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MDPI AG
2025-07-01
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| author | Helin Savsin Tomasz Tokarek |
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| description | Hypertrophic cardiomyopathy (HCM) is the most common monogenic heart disease, with an estimated prevalence of 1:600 in the general population, and is associated with significant morbidity. HCM is characterized by left ventricular hypertrophy and interventricular septal thickening due to sarcomere protein gene mutations. The recent emergence of cardiac myosin inhibitors (CMIs), specifically mavacamten and aficamten, has introduced a paradigm shift in HCM management by directly targeting the hypercontractile state of the disease. This review comprehensively discusses the molecular mechanisms of mavacamten and aficamten, highlighting their biochemical similarities and differences from available data. It evaluates their reported efficacy in completed clinical trials, such as reducing left ventricular outflow tract (LVOT) obstruction, improving functional capacity, and enhancing quality of life in HCM. It further provides insight and updates to ongoing trials of both CMIs. Finally, it compares and elaborates on the safety profiles of mavacamten and aficamten, discussing their favorable safety profiles shown in completed studies. In current clinical practice, only mavacamten is approved for use, and clinical insights concerning both CMIs are limited, but encouraging. In summary, cardiac myosin inhibitors are a promising class of disease-modifying drugs for HCM with proven short-term safety and efficacy, but limited data are available to fully determine their long-term effects and efficacy in diverse patient populations. Ongoing research is necessary to further explore and define their role in HCM management. |
| format | Article |
| id | doaj-art-94e5a4a43e2a41e58e68147004103a69 |
| institution | DOAJ |
| issn | 2227-9059 |
| language | English |
| publishDate | 2025-07-01 |
| publisher | MDPI AG |
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| series | Biomedicines |
| spelling | doaj-art-94e5a4a43e2a41e58e68147004103a692025-08-20T03:02:48ZengMDPI AGBiomedicines2227-90592025-07-01137161910.3390/biomedicines13071619Comprehensive Review: Mavacamten and Aficamten in Hypertrophic CardiomyopathyHelin Savsin0Tomasz Tokarek1Center for Innovative Medical Education, Jagiellonian University Medical College, Medyczna 9, 31-008 Krakow, PolandCenter for Innovative Medical Education, Jagiellonian University Medical College, Medyczna 9, 31-008 Krakow, PolandHypertrophic cardiomyopathy (HCM) is the most common monogenic heart disease, with an estimated prevalence of 1:600 in the general population, and is associated with significant morbidity. HCM is characterized by left ventricular hypertrophy and interventricular septal thickening due to sarcomere protein gene mutations. The recent emergence of cardiac myosin inhibitors (CMIs), specifically mavacamten and aficamten, has introduced a paradigm shift in HCM management by directly targeting the hypercontractile state of the disease. This review comprehensively discusses the molecular mechanisms of mavacamten and aficamten, highlighting their biochemical similarities and differences from available data. It evaluates their reported efficacy in completed clinical trials, such as reducing left ventricular outflow tract (LVOT) obstruction, improving functional capacity, and enhancing quality of life in HCM. It further provides insight and updates to ongoing trials of both CMIs. Finally, it compares and elaborates on the safety profiles of mavacamten and aficamten, discussing their favorable safety profiles shown in completed studies. In current clinical practice, only mavacamten is approved for use, and clinical insights concerning both CMIs are limited, but encouraging. In summary, cardiac myosin inhibitors are a promising class of disease-modifying drugs for HCM with proven short-term safety and efficacy, but limited data are available to fully determine their long-term effects and efficacy in diverse patient populations. Ongoing research is necessary to further explore and define their role in HCM management.https://www.mdpi.com/2227-9059/13/7/1619mavacamtenaficamtencardiomyopathycardiac myosin inhibitorsdisease-modifying therapyleft ventricular hypertrophy |
| spellingShingle | Helin Savsin Tomasz Tokarek Comprehensive Review: Mavacamten and Aficamten in Hypertrophic Cardiomyopathy Biomedicines mavacamten aficamten cardiomyopathy cardiac myosin inhibitors disease-modifying therapy left ventricular hypertrophy |
| title | Comprehensive Review: Mavacamten and Aficamten in Hypertrophic Cardiomyopathy |
| title_full | Comprehensive Review: Mavacamten and Aficamten in Hypertrophic Cardiomyopathy |
| title_fullStr | Comprehensive Review: Mavacamten and Aficamten in Hypertrophic Cardiomyopathy |
| title_full_unstemmed | Comprehensive Review: Mavacamten and Aficamten in Hypertrophic Cardiomyopathy |
| title_short | Comprehensive Review: Mavacamten and Aficamten in Hypertrophic Cardiomyopathy |
| title_sort | comprehensive review mavacamten and aficamten in hypertrophic cardiomyopathy |
| topic | mavacamten aficamten cardiomyopathy cardiac myosin inhibitors disease-modifying therapy left ventricular hypertrophy |
| url | https://www.mdpi.com/2227-9059/13/7/1619 |
| work_keys_str_mv | AT helinsavsin comprehensivereviewmavacamtenandaficamteninhypertrophiccardiomyopathy AT tomasztokarek comprehensivereviewmavacamtenandaficamteninhypertrophiccardiomyopathy |