NDA for First-in-Class DMD Tx Submitted to FDA
PTC Therapeutics announced the completion of their rolling submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
The FDA has previously granted Translarna Orphan Drug status for DMD, cystic fibrosis (CF), Mucopolysaccharidosis I (MPS I), and aniridia.
Translarna is an investigational, first-in-class, oral protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation such as in nmDMD.
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