PTC Therapeutics (PTCT) Granted FDA 10/24/17 PDUFA Date for Translarna

March 6, 2017 8:03 AM EST

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PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has acknowledged the filing over protest of PTC's New Drug Application (NDA) for Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The Company is seeking approval to market the drug for the treatment of nmDMD patients in the United States. Translarna received marketing authorization for patients with nmDMD in the European Union in August 2014 and is now available in over 25 countries.

The FDA has granted standard review and assigned a Prescription Drug User Fee Act (PDUFA) date of October 24, 2017. The PDUFA date is the target date for the FDA to complete its review of the NDA.

"We look forward to working closely with the FDA and the DMD community to bring this much-needed therapy to patients," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We believe that the totality of clinical data in our NDA, which includes the results of two of the largest placebo-controlled DMD clinical trials ever conducted, demonstrates Translarna's benefits to patients and merits a full and fair review by the FDA, including an advisory committee meeting."

Primarily affecting males, Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD lose the ability to walk in their early teens and experience life-threatening lung and heart complications in their late teens and twenties. It is estimated that nonsense mutations account for approximately 13% of DMD cases.

PTC used the FDA's file over protest regulations to file the NDA. These regulations allow a company to have its NDA filed and reviewed following receipt of a refuse to file determination.



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