New drug for hypertrophic cardiomyopathy wins special support in America

5th May 2016

A drug being developed to treat the underlying cause of hypertrophic cardiomyopathy (HCM) has won special support in America.

The drug, termed MYK-461, has been granted orphan drug designation by the Food and Drug Administation.

Being developed by the biopharmaceutical company MyoKardia, the drug is for the treatment of obstructive hypertrophic cardiomyopathy.  This is when the thickened heart muscle obstructs blood flow out of the heart, causing symptoms.

The drug is designed to reduce excessive heart muscle contractility and the company says the therapy is the first designed to treat the underlying cause of HCM.

The orphan drug designation, which supports the development of medicines in underserved or rare disorders, gives the company various financial benefits.

Chief executive Tassos Gianakakos said: “This represents another step toward filling a critical need for patients and families who struggle with a chronic and debilitating illness for which there are no approved therapies.”

Trials have started to assess the drug’s ability to reduce excessive heart muscle contractility

The company reports that data from two of the phase 1 trials demonstrated clinical proof of the mechanism in both HCM patients and healthy volunteers, and is well tolerated.

HCM with obstruction can cause symptoms including shortness of breath and chest pain. Patients are typically prescribed one or more drugs (including beta-blockers, calcium channel blockers and disopyramide) indicated for the treatment of hypertension, heart failure or other cardiovascular disorders more generally. Those with more severe disease or more symptoms, may require surgery or other invasive interventions.

For more information about the drug, see here.