Posted by Michael Wonder on 24 Jul 2020
MyoKardia announces receipt of breakthrough therapy designation from FDA for mavacamten for the treatment of symptomatic, obstructive hypertrophic cardiomyopathy
23 July 2020 - MyoKardia today announced that the U.S. FDA has granted Breakthrough Therapy Designation to mavacamten, a novel, oral, allosteric modulator of cardiac myosin, for the treatment of symptomatic, obstructive hypertrophic cardiomyopathy.
In May, MyoKardia announced top-line results from the company’s pivotal Phase 3 EXPLORER clinical trial of mavacamten for the treatment of symptomatic patients with obstructive HCM. Mavacamten was well tolerated, with a safety profile comparable to placebo, and demonstrated a robust treatment effect, with patients experiencing clinically meaningful responses to treatment, including reductions in symptoms, improvements in cardiac function and reduction or elimination of the obstruction of the left ventricle. MyoKardia is currently preparing a new drug application for mavacamten, with plans to submit to the FDA in the first quarter of 2021.
