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MyoKardia Gets Orphan Drug Designation For MYK-461 For Treatment Of OHCM

MyoKardia, Inc. (MYOK), a clinical stage biopharmaceutical company for the treatment of heritable cardiovascular diseases, announced Monday that the U.S. Food and Drug Administration has granted the company Orphan Drug Designation for MYK-461 for treatment of symptomatic obstructive hypertrophic cardiomyopathy or oHCM, a subset of hypertrophic cardiomyopathy or HCM.

MYK-461, an orally administered small molecule, is the Company's lead product candidate and the first therapy designed to target the underlying cause of HCM. MyoKardia's proposed initial indication for MYK-461 is oHCM.

Three Phase 1 clinical trials have been initiated to assess MYK-461's ability to modulate cardiac myosin by measuring reduction in cardiac muscle contractility via echocardiography.

The FDA Office of Orphan Products Development grants orphan status to support development of medicines for underserved patient populations, or rare disorders that affect fewer than 200,000 people in the United States.

The company noted that orphan drug designation provides it with certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

There are currently no approved therapeutic products indicated for the treatment of HCM. Patients are typically prescribed one or more drugs indicated for the treatment of hypertension, heart failure or other cardiovascular disorders more generally.

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