Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for mavacamten, an investigational, novel, oral, allosteric modulator of cardiac myosin, for patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of January 28, 2022.
HCM, which is the most common inherited heart disease, can be a chronic, debilitating, and progressive condition where patients may experience symptoms of shortness of breath, dizziness and fatigue as well as serious, life-altering complications, including heart failure, arrhythmias, stroke and sudden cardiac death,” said Roland Chen, M.D., Senior Vice President, Cardiovascular Development, Bristol Myers Squibb. “Today’s acceptance from the FDA puts us one step closer to having a highly targeted therapeutic approach for oHCM, as mavacamten is a first-in-class myosin inhibitor developed to address the underlying molecular defect of the disease. We are committed to supporting patients in need of HCM treatment and look forward to working with the FDA.”
The NDA submission was based on the results of the pivotal Phase 3 EXPLORER-HCM trial, which evaluated mavacamten in patients with symptomatic oHCM versus placebo. Results from the trial showed that mavacamten demonstrated a robust treatment effect, with clinically meaningful improvements in symptoms, functional status, and quality of life, as well as the ability to relieve left ventricular outflow tract obstruction. In the EXPLORER-HCM study all primary and secondary endpoints were met with statistical significance.
Mavacamten is an investigational therapy that is not approved for use in any country.