logo
Share SHARE
FONT-SIZE Plus   Neg

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.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
KMG Chemicals Inc. (KMG) is likely to beat earnings per share or EPS estimates, when it reports first quarter results on December 11, after the bell. First quarter results of KMG are expected to benefit from the impact of Irma and Harvey, and early October fires in the West. Sysco Corp. is looking ahead to own 50 fully electric semi tracks to be launched by Tesla. The new electric tractors could run 500 miles on a single charging. The order from the food service distribution giant that owns more than 7000 trucks is worth up to $10 million. Patreon, the crowdfunding platform for artists, announced Wednesday a change in its payment structure that it says will help creators keep more money. Meanwhile, many are concerned that the change may discourage their patrons from pledging.
comments powered by Disqus
Follow RTT