PTC Therapeutics Begins Rolling NDA Submission To FDA For Translarna

(RTTNews) - Biopharmaceutical company PTC Therapeutics, Inc. (PTCT) announced that it has commenced a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation, which is an alteration in the genetic code that prematurely halts the synthesis of an essential protein.

A rolling submission allows completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis.

PTC expects to finalize the application in the fourth quarter of 2015 following the completion of the ACT DMD confirmatory Phase 3 clinical trial.