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MyoKardia Readies First-Ever Hypertrophic Cardiomyopathy Drug For US Filing

 
• By Mandy Jackson

The Phase III EXPLORER-HCM trial evaluating mavacamten in obstructive hypertrophic cardiomyopathy (oHCM) succeeded on all primary and secondary endpoints.

heart surround with pharmaceutical medicine pills and tablets, a flask and a pill box on blue background, heart health and treatment. top view. health concept
MyoKardia's once-daily pill bested placebo on all efficacy measures • Source: Shutterstock

MyoKardia Inc. plans to file mavacamten with the US Food and Drug Administration in the first quarter of 2021 based on results from the Phase III EXPLORER-HCM clinical trial in obstructive hypertrophic cardiomyopathy (oHCM). The company reported on 11 May that patients treated with the oral drug were less likely to experience shortness of breath than those who received placebo in the pivotal trial, and half of the patients treated with mavacamten were deemed asymptomatic by the end of the study.

The EXPLORER-HCM results show that the allosteric modulator of cardiac myosin improved patients’ heart function and symptoms while proving at...

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