Mavacamten for treating non-obstructive hypertrophic cardiomyopathy

Mavacamten is in clinical development for treatment of non-obstructive hypertrophic cardiomyopathy (HCM). HCM is often an inherited disease, where the heart muscle cells enlarge, and the walls of the heart thicken. If there is no significant blocking of blood flow, the condition is called nonobstructive HCM (nHCM). This is caused by excessive binding of myosin and actin (proteins involved in muscle contraction) in the heart muscle, which leads to abnormally increased contractions in the heart. This affects the hearts’ ability to pump blood around the body effectively, and can cause symptoms including dizziness, fatigue, heart murmurs and swelling of legs, ankles and/or abdomen. Patients with nHCM are at risk of serious conditions such as sudden cardiac death and stroke. Currently, treatment strategies for nHCM are aimed at managing symptoms, and there are no licensed therapies available for treating the underlying cause of disease.