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EMA Validates Bristol Myers Squibbs Application for Mavacamten

  • If approved, mavacamten would be the first cardiac myosin inhibitor for the treatment of obstructive hypertrophic cardiomyopathy.
  • Application based on positive results from Phase 3 EXPLORER-HCM trial.

Bristol Myers Squibb today announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for mavacamten, an investigational, first-in-class cardiac myosin inhibitor, for the treatment of patients with obstructive hypertrophic cardiomyopathy (obstructive HCM). 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 3 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.

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About the Phase 3 EXPLORER-HCM Trial
The EXPLORER-HCM Phase 3 trial enrolled a total of 251 patients with symptomatic (NYHA Class II or III) obstructive hypertrophic cardiomyopathy. All participants had measurable left ventricular outflow tract (LVOT) gradient (resting and/or provoked) ≥50 mmHg at baseline.

The primary endpoint for EXPLORER-HCM was a composite functional analysis designed to capture mavacamten’s effect on both symptoms and function. Secondary endpoints were changes from baseline to week 30 in post­exercise LVOT gradient, pVO2, proportion of patients with at least one NYHA class improvement, and measures of patient­ reported outcomes. Additional endpoints included changes from baseline to Week 30 in echocardiographic indices, circulating biomarkers, cardiac rhythm patterns and accelerometry.

About Obstructive Hypertrophic Cardiomyopathy
Obstructive hypertrophic cardiomyopathy (obstructive HCM), the most common type of HCM, is a chronic, progressive disease in which the heart muscle becomes abnormally enlarged or thick causing the left ventricular outflow tract (LVOT) where blood leaves the heart to become obstructed by the enlarged heart muscle. As a result, obstructive HCM can lead to debilitating symptoms for patients and has also been associated with increased risks of atrial fibrillation, stroke, heart failure and sudden cardiac death.

The most frequent cause of obstructive HCM is mutations in the heart muscle proteins of the sarcomere and as many as 50% of patients have a hereditary predisposition to the disease. Obstructive HCM is estimated to affect 400,000-600,000 people worldwide, however many patients remain undiagnosed and/or asymptomatic.

About Mavacamten
Mavacamten is a first-in-class, oral, allosteric modulator of cardiac myosin being investigated for the treatment of conditions caused by excessive cardiac contractility and impaired diastolic filling of the heart, including hypertrophic cardiomyopathy (HCM) and heart failure with preserved ejection fraction (HFpEF).

In clinical studies, mavacamten has demonstrated significant efficacy in reducing cardiac muscle contractility by reducing excess actin-myosin cross-bridging, leading to less hypercontractility and improved relaxation.

In obstructive HCM specifically, it is the first and only selective cardiac myosin inhibitor that has the potential to treat the underlying pathophysiology of the disease.

Mavacamten is an investigational therapy and is not approved for use in any country.

Source: Bristol-Myres Squibb
Date: Oct 1, 2021

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