FDA Approves Bristol Myres’ Drug to Treat Inherited Heart Condition

The Food and Drug Administration has approved Bristol Myers Squibb Camzyos, the first drug to treat the cause of hereditary heart failure known as obstructive hypertrophic cardiomyopathy.

Obstructive hypertrophic cardiomyopathy is a condition in which the heart muscle thickens and affects its ability to pump blood. Patients often present with symptoms such as heart failure, and shortness of breath, and have been treated with beta-blockers and calcium channel blockers as well.

Camzyos was approved based on a study of 251 patients called EXPLORER. In this study, patients randomized to take the drug had significantly improved peak oxygen consumption and improved widely used heart failure measurements compared to patients receiving a placebo. Hirawat pointed out that in 27% of patients taking medications that "return to normal" the symptoms of heart failure refer to the symptoms on the scale.

According to a company spokesman, the drug is priced at $89,500 for a year of treatment. This figure far exceeds the $12,000 to $15,000 annual pricing benchmark set by the Institute for Clinical and Economic Review, an influential group for assessing the cost-effectiveness of medicines. ICER has concluded based on the additional benefits of Camzyos over a drug called disopyramide.

The approval also included a warning about the risk of heart failure and an FDA-mandated plan to manage that risk because Camzyos relaxes the myocardium, patients may experience a decrease in "ejection fraction" or a decrease in the volume of blood in the left ventricle of the heart that is pumped into the arteries.