Pharma Focus Asia

Novel Mavacamten Discovered to Treat Obstructive Hypertrophic Cardiomyopathy (oHCM)

LianBio, discovered novel Mavacamten for the treatment of patients with obstructive hypertrophic cardiomyopathy (oHCM).

Mavacamten is a first-in-class, oral, allosteric modulator of cardiac myosin which is under investigation for the treatment of excessive cardiac contractility. It lowers the risk of cardiac muscle contractility by restricting the excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance.

In EXPLORER-HCM trial, mavacamten succeeded in all primary and secondary endpoints and evaluated significant improvements in functional status, symptoms, and quality of life.

It is estimated that approximately 1.1 million1 to 2.8 million2 people are affected with HCM in China, having no effective drug treatment options beyond limited symptomatic relief treatment options.

Hypertrophic cardiomyopathy or HCM is a chronic disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can lead to the development of debilitating symptoms and cardiac dysfunction. Globally, it is estimated that one in every 500 people are effected with HCM.

HCM is mainly caused due to mutations in the heart muscle proteins of the sarcomere. Moreover, both obstructive or non-obstructive HCM patients results in fatigue or shortness of breath which intefers the patient’s day-to-day activities.

Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) grants Breakthrough Therapy Designation for mavacamten to treat obstructive hypertrophic cardiomyopathy (oHCM).

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