Milind Desai, MD, MBA, director of the Center for Hypertrophic Cardiomyopathy and medical director of the Center for Aortic Diseases, Cleveland Clinic, speaks on therapeutic implications of the VALOR-HCM late-breaking clinical trial findings presented Saturday at the American College of Cardiology's 71st Scientific Session.
The myosin inhibitor mavacamten is the first therapy to show long-term reduction in gradient, improvement in quality of life, and improvement in functional capacity in the treatment of hypertrophic cardiomyopathy (HCM), said Milind Desai, MD, MBA, director of the Center for Hypertrophic Cardiomyopathy and medical director of the Center for Aortic Diseases, at Cleveland Clinic.
How are emerging pharmacological options for obstructive hypertrophic cardiomyopathy advancing treatment of the disease compared with established drugs?
Established drugs were never developed for specific management of HCM. In fact, I will tell you, earlier this year in 2022 or maybe late 2021, there was a paper published in Journal of the American College of Cardiology looking at a small group of patients randomized to beta-blockers vs not, and that showed that it reduces gradient, but it did not show an improvement in functional capacity. All it showed was reduction in gradient.
This small prospective study is one of the 2 that I know, of prospective studies. The last prospective study on a beta-blocker was developed in the late 60s, early 70s, and that was also in a handful of patients. There is no prospective trial on calcium-channel blockers. There is no prospective trial on disopyramide.
Essentially, the previous data, as a serendipity, is how we have started using the other drugs. The myosin inhibitors is the first therapy that has been tested in a randomized control trial in a multicenter format, with a long-term extension arm that has shown reduction in gradient, improvement in quality of life, and improvement in functional capacity.