Press Release

Disclosures: Gianakakos is an employee of MyoKardia.
July 24, 2020
1 min read

FDA grants breakthrough therapy designation to mavacamten


Press Release

Disclosures: Gianakakos is an employee of MyoKardia.
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MyoKardia announced that the FDA has granted breakthrough therapy designation to mavacamten for the treatment of patients with symptomatic, obstructive hypertrophic cardiomyopathy.

Mavacamten is a novel, oral, allosteric modulator of cardiac myosin, according to a press release from the company.

FDA HQ in Washington
Source: Adobe Stock.

“Receipt of breakthrough therapy designation from the FDA acknowledges that both the significant unmet need among patients with obstructive [hypertrophic cardiomyopathy], for whom there are currently no targeted therapies, and the highly encouraging clinical results generated by mavacamten,” Tassos Gianakakos, CEO of MyoKardia, said in the release.

The company announced top-line results of the EXPLORER-HCM trial in May. As Healio previously reported, the trial met its primary endpoint, a composite functional analysis to assess the effect of mavacamten on symptoms and function compared with placebo (P = .0005), in addition to secondary endpoints including improvements in NYHA functional classification (P < .0001), post-exercise left ventricular outflow tract peak gradient (P < .0001), Kansas City Cardiomyopathy Clinical Summary Score (P < .0001), peak VO2 (P = .0006) and the HCM Symptom Questionnaire Shortness of Breath Domain Score (P < .0001).

“We look forward to working closely with the FDA in pursuit of regulatory approval so that we may bring mavacamten to people with [hypertrophic cardiomyopathy] and remain appreciative of the collaborative approach and valued input the agency has provided through mavacamten’s development,” Gianakakos said in the release.

The company is currently preparing a new drug application for the drug and plans to submit it in the first quarter of 2021, according to the release.