Mavacamten（MYK-461）is a first-in-class allosteric inhibitor of cardiac myosin that promises to provide clinicians with targeted therapy for these patients. It is a mechanistically novel small molecule that acts on the sarcolemma to specifically inhibit contractility and has been proposed as a treatment for HCM.
MYK-461 is a drug used to treat obstructive hypertrophic cardiomyopathy as a cardiac myosin inhibitor. Mavacamten is used to treat adults with symptomatic obstructive hypertrophic cardiomyopathy. It is used to treat symptomatic obstructive hypertrophic cardiomyopathy (HCM).
Mavacamten is thought to work by reducing cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance. In clinical and preclinical studies, it has consistently reduced biomarkers of cardiac wall stress, lessened excessive cardiac contractility, and increased diastolic compliance.
Common side effects include:Dizziness, fainting, Incidence not known, Chest pain or tightness, decreased urine output, dilated neck veins, irregular breathing, irregular heartbeat, swelling of the face, fingers, feet, or lower legs, trouble breathing, unusual tiredness or weakness and weight gain.
