Dr Jay Edelberg Outlines the Clinical Burdens and Treatment Options for Hypertrophic Cardiomyopathy

November 17, 2019

Hypertrophic cardiomyopathy can be a debilitating condition that currently has no approved therapies to treat it, highlighting the impact of potential treatments like mavacamten, said Jay Edelberg, MD, PhD, senior vice president of Clinical Development at MyoKardia.

Hypertrophic cardiomyopathy can be a debilitating condition that currently has no approved therapies to treat it, highlighting the impact of potential treatments like mavacamten, said Jay Edelberg, MD, PhD, senior vice president of Clinical Development at MyoKardia.

Transcript

What are the clinical and cost burdens of hypertrophic cardiomyopathy on the U.S. health system? Is the burden from this condition increasing?

Hypertrophic cardiomyopathy is a fairly common form of cardiomyopathy—it affects 1 in 500 people in the United States and globally. Hypertrophic cardiomyopathy manifests in a variety of ways for patients. Some people are able to get by with minimal symptoms through a large part of their life. For others, they’re impacted by heart failure, atrial fibrillation, ventricular fibrillation, sudden cardiac death, worsening heart failure–so, it really actually presents in a variety of ways. For some people, they’re just limited by activities of daily living. For others, it actually can really be quite debilitating, and their only options end up being surgery or even heart transplantation.

With the recent JAMA Cardiology study showing an increase in heart failure in the US population, where does mavacamten fit into the treatment paradigm?

Mavacamten is being developed for obstructive hypertrophic cardiomyopathy—we’re in phase 3 in that study. So, this is a sub-form of heart failure–so it’s patients with heart failure that’s due to actually the heart actually working in overtime. It’d be like basically your bicep muscles having to work 24 hours a day, 7 days a week every day of your life. They just become so very big, thick, and frankly so non-compliant that actually they don’t end up working very well–the heart is not able to fill well with blood and it ends up actually leading to a heart failure for those patients there. So, it’s a very specialized form of heart failure. We think that the molecular mechanisms of hypertrophic cardiomyopathy may actually give us insight how to treat some patients with heart failure with preserved ejection fraction. It makes up approximately half of all of heart failure in the US, and we presently don’t have any therapies that are approved for that.