logo
Plus   Neg
Share
Email

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 RTTNews Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
A former Southwest Airlines employee has filed a racial discrimination lawsuit against the airline, alleging that the carrier allowed its employees to create a "whites-only" breakroom. The lawsuit was filed in the United States District Court in Houston earlier this week by Jamel Parker, a former employee of Southwest Airlines. American Airlines Inc. has joined its major rivals in raising baggage fees as the airline industry grapples with higher fuel costs and rising operating expenses. The largest U.S. airline said Thursday it is raising the price of a first and second checked bag by $5 each. The price of a first checked bag is now $30, while it is $40 for a second checked bag. The takeover battle by Comcast Corp. nd Rupert Murdoch's 21st Century Fox Inc. for British broadcaster Sky plc will be settled through an auction. The UK Takeover Panel said Thursday that an auction procedure is expected to commence on the evening of Friday, September 21, 2018 and end on the evening of September 22, 2018. The winner of the auction will be announced on Saturday.
Follow RTT