FDA grants breakthrough therapy designation to mavacamten
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.
“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.