FDA Approves Mavacamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy

The first in-class myosin inhibitor is supported by findings from the pivotal EXPLORER-HCM trial for patients with the rare condition.

The US Food and Drug Administration (FDA) has approved mavacamten (Camzyos) for the treatment of obstructive hypertrophic cardiomyopathy (oHCM).

The first in-class myosin inhibitor is designed to decrease contractile function and improve VO2 in patients with the rare inherited disease that often results in dynamic left ventricular outflow tract (LVOT) obstruction.

Mavacamten now becomes the first allosteric and reversible selective cardiac myosin inhibitor designed to target underlying pathophysiology of oHCM granted indication by the FDA. Its approval was granted to Bristol Myers Squibb.

Phase 3 trial data from the EXPLORER-HCM supported the drug's benefit. In the trial, patients with LVOT ≥50 mmHg and New York Heart Association class II-III symptoms reported sustained improvement in questionnaire summary scores for oHCM with mavacamtean versus placebo over 30 weeks.

Late-breaking findings from the EXPLORER-HCM extension study presented at the American College of Cardiology (ACC) 2022 Scientific Sessions last month found the use of mavacamten had continual and consistent benefits in quality of life and outcomes over the long-term in interim results.

During an interview with HCPLive, study presenter Florian Rader, MD, Co-Director, Clinic for Hypertrophic Cardiomyopathy and Aortopathies, Cedars-Sinai, expressed his perspective on the significance of this data and the medication class.

"Since HCM was first described, more than 60 years ago, there really hasn't been any specific targeted drug therapy for this disease and mavacamten is now really the most promising candidate for this role,” he said.

Rader additionally discussed what the treatment uptake might look like pending its approval.

"I think like with any new medication class, there will probably be larger HCM centers that will adopt this new medication probably a little bit more quicker than smaller practices," he said. "And it will also probably depend on how stringent the safety monitoring requirements will be for this new medication and price might also play a role."

In a statement accompanying the approval, Milind Desai, MD, MBA, director of the Hypertrophic Cardiomyopathy Center and director of clinical operations in Cleveland Clinic’s Heart Vascular & Thoracic Institute, highlighted the significance of this novel drug approval.

“This is a first-in-class medicine specifically for patients living with symptomatic obstructive HCM,” Desai said. "With this FDA approval, US cardiologists now have a new pharmacological option for eligible patients that targets the underlying pathophysiology of the disease.”

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