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Stock Market & Financial Investment News

News Breaks
July 9, 2014
14:01 EDTPTCTPTC Therapeutics announces expanded access program for Translarna
PTC Therapeutics announced the initiation of a reimbursed expanded access program, or EAP. PTC's EAP program is intended to make Translarna available to patients before commercial availability in certain countries. Where mechanisms exist and in accordance with local regulations, PTC will make Translarna available to nonsense mutation Duchenne muscular dystrophy patients through funded EAP programs.
News For PTCT From The Last 14 Days
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February 8, 2016
10:17 EDTPTCTSarepta approval seen as 'long shot' as FDA pushes back decision
Shares of drugmaker Sarepta Therapeutics (SRPT) took a dive in morning trading after the company said the U.S. Food and Drug Administration delayed a decision on eteplirsen for the treatment of Duchenne muscular dystrophy. WHAT'S NEW: Sarepta announced that the FDA has pushed out the the Prescription Drug User Fee Act, or PDUFA, date for eteplirsen for the treatment of Duchenne muscular dystrophy to May 26. The previous PDUFA date for the eteplirsen new drug application, or NDA, was originally set for late February. FDA POSTPONEMENT: The FDA notified Sarepta that it's submission of 4-year clinical data, including additional six minute walk test and loss of ambulation data compared to historical control, was designated as a "major amendment" to the NDA and that the PDUFA goal date was extended to allow for a full review of the submission. The FDA previously granted eteplirsen Priority Review status, as well as Rare Pediatric Disease Designation, Orphan Drug Designation and Fast Track Status to eteplirsen. WHAT'S NOTABLE: In late January, the FDA postponed a meeting of its advisory committee to review the Sarepta drug due to an inclement weather forecast for Washington D.C. LONG SHOT: Piper Jaffray analyst Edward Tenthoff told investors in a note that the FDA's PDUFA date postponement for eteplirsen is "not surprising" following the delay of the January 22 AdCom due to weather. Tenthoff also noted that a new AdCom has yet to be scheduled. The analyst continues to see eteplirsen accelerated approval by the new PDUFA date as a "long shot." He believes that the panel discussion will be a "plea from the community" to approve something for DMD and that members will not be asked to vote on drug approval. Tenthoff has a Neutral rating and $15 price target on Sarepta. OTHERS TO WATCH: Other companies developing treatments for Duchenne muscular dystrophy include BioMarin (BMRN) and PTC Therapeutics (PTCT). PRICE ACTION: Sarepta is down 9.5% in morning trading to $11.06.

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