FDA approves 1st drug for hypertrophic cardiomyopathy

The FDA on April 28 approved Bristol Myers Squibb's Camzyos, the first drug to target the cause of obstructive hypertrophic cardiomyopathy. 

The disease causes the heart muscle to thicken, which leads to forceful contractions and makes it harder for the organ to pump blood. It often goes undiagnosed since many people have few, if any, symptoms. For those with symptoms, shortness of breath and chest pain are among the most common. 

The drug, which goes by the chemical name mavacamten, was approved for adults with symptoms of the condition. Camzyos blocks myosin, a protein believed to play a key role in causing forceful contractions, The Wall Street Journal reports. 

"This is a first-in-class medicine specifically for patients living with symptomatic obstructive HCM," said Milind Desai, MD, director of the Hypertrophic Cardiomyopathy Center and director of clinical operations at Cleveland Clinic's Heart Vascular & Thoracic Institute. "With this FDA approval, U.S. cardiologists now have a new pharmacological option for eligible patients that targets the underlying pathophysiology of the disease." 

The approval is based on phase 3 trial results involving 251 participants. Compared to those who took a placebo, patients with HCM who took Camzyos had improved heart function, quality of life and oxygen consumption after 30 weeks. While no serious safety concerns were observed, the approval came with a boxed warning for heart failure, as the drug reduces left ventricular ejection fraction and can cause heart failure due to systolic dysfunction. It is only available under an FDA-required program called Risk Evaluation and Mitigation Strategy.

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