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PTC Therapeutics Inc announces US FDA tentative meeting on 28 September 2017 to review NDA for ataluren.

M2 EQUITYBITES-June 7, 2017-PTC Therapeutics Inc announces US FDA tentative meeting on 28 September 2017 to review NDA for ataluren


Biopharmaceutical company PTC Therapeutics Inc (Nasdaq:PTCT) said on Tuesday that the US Food and Drug Administration (FDA) has announced the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting for 28 September 2017 to review the new drug application (NDA) for ataluren.

According to the company, ataluren (Translarna) is an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation dystrophinopathy, including nonsense Duchenne muscular dystrophy (nmDMD).

A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy, added the company.

In conjunction, the company's NDA submission for ataluren was awarded standard review by the US FDA in March 2017. The US FDA set a Prescription Drug User Fee Act (PDUFA) goal date of 24 October 2017 for completion of its review of the ataluren NDA.

Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein.

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Publication:M2 EquityBites (EQB)
Date:Jun 7, 2017
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