European Medicines Agency validates MAA for mavacamten for the treatment of obstructive hypertrophic cardiomyopathy.- BMS.

Validation of the application confirms the submission is complete, and the EMA’s centralized procedure with Committee for Medicinal Products for Human Use (CHMP)’s assessment begins.

“Despite the global prevalence of obstructive HCM and its debilitating symptoms and cardiac dysfunction, there is yet to be an approved therapy that targets the underlying cause of this devastating disease. Currently prescribed medicines largely provide only symptom relief. Mavacamten could potentially provide a treatment option that addresses the unmet needs of people living with obstructive HCM around the world,” said Roland Chen, M.D., senior vice president, Cardiovascular Development, Bristol Myers Squibb. “Today’s acceptance of the dossier by the EMA is a step forward in bringing this important targeted therapeutic approach to patients and physicians in Europe, and we thank the patients and investigators who have been involved in the EXPLORER-HCM trial.”

The application is based on the results of the pivotal Phase III EXPLORER-HCM trial, which evaluated mavacamten in patients with symptomatic obstructive HCM versus placebo. Results from the trial showed mavacamten demonstrated a clear 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.