The extension will give the FDA sufficient time to look into the drug for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
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The U.S. Food and Drug Administration (FDA) has extended its review of Bristol Myers Squibb‘s New Drug Application (NDA) for the proposed heart drug mavacamten to allow more time to evaluate its product information.
The extension to April 28, 2022, will give the regulator sufficient time to look into the drug for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The FDA will especially evaluate updates to the proposed Risk Evaluation Mitigation Strategy (REMS), which was included in the initial application.
Mavacamten is a candidate cardiac myosin inhibitor for patients diagnosed with oHCM. The company got ahold of mavacamten in November last year, following its $13.1 billion acquisition of MyoKardia. The NDA follows positive results from the Phase III EXPLORER-HCM study, which demonstrated an improvement in health status in patients after 30 weeks compared to those who took a placebo.
Outcomes were measured using the 23-item Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ OSS), where 36% exhibited at least five points of change versus the 15% from the placebo group. The questions evaluated symptoms, social and physical function, and quality of life.
The primary endpoint for the trial was a composite functional analysis designed to assess mavacamten’s effect on symptoms and function. The secondary endpoints were baseline changes at week 30 in pVO2, postexercise LVOT gradient, number of patients reporting improvement in at least one NYHA class, and patient reported outcomes. Other endpoints included week 30 baseline changes in circulating biomarkers, accelerometry, echocardiographic indices, and cardiac rhythm patterns.
“We are confident in the profile of mavacamten. This first-in-class cardiac myosin inhibitor demonstrated clinically meaningful improvements in symptoms, functional status, and quality of life in symptomatic oHCM patients in the pivotal EXPLORER-HCM trial. We look forward to continuing to work closely with the FDA to bring this important medicine to patients,” said Samit Hirawat, M.D., the executive vice president and chief medical officer for global drug development for Bristol Myers Squibb, in a statement.
HCM is a chronic, progressive disease characterized by an excessive contraction of the heart muscle and reduced ability of the left ventricle to fill. It affects one in every 500 persons and can lead to cardiac dysfunction and other debilitating symptoms.
There are around 160,000 to 200,000 people diagnosed with the disease in the U.S. and the E.U., and there are no existing effective treatment options beyond providing relief for symptoms. HCM is also not always diagnosed, with only 25% of those with obstructive HCM and only 10% of those with non-obstructive HCM receiving a proper diagnosis.
The FDA has, so far, not requested any other documentation or studies to support its review extension.
