The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for mavacamten, a novel therapy for symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Mavacamten is an oral, selective allosteric inhibitor of cardiac myosin. It reduces cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance.
The NDA submission was based on data from the phase 3 EXPLORER-HCM trial [ClinicalTrials.gov: NCT03470545], which evaluated mavacamten vs placebo in 251 adult patients with symptomatic, obstructive hypertrophic cardiomyopathy. Results showed clinically meaningful improvements in symptoms, functional status, and quality of life among patients treated with mavacamten.
“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,” said Roland Chen, MD, Senior Vice President, Cardiovascular Development, Bristol Myers Squibb.
A Prescription Drug User Fee Act (PDUFA) date of January 8, 2022 has been set for the application review.
U.S. Food and Drug Administration (FDA) accepts Bristol Myers Squibb’s application for mavacamten in symptomatic obstructive hypertrophic cardiomyopathy (oHCM). [press release]. Princeton, NJ: Bristol Myers Squibb; March 19, 2021.