The Office of Drug Evaluation of the FDA rejects an appeal from PTC Therapeutics against the refusal to file letter from FDA for Translarna (ataluren) to treat Duchenne muscular dystrophy.

PTC Therapeutics announced that at the end of last week, the Office of Drug Evaluation I (ODE-I) of the FDA denied the company's first appeal of the refuse to file letter issued by the FDA's Division of Neurological Products (DNP) on February 22, 2016 regarding PTC's New Drug Application (NDA) for Translarna (ataluren)for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The company intends to escalate its appeal to the next supervisory level of the FDA. This is an iterative process and the company anticipates that multiple cycles of appeals to progressively higher levels of the FDA may be required.

The European Medicines Agency granted under a special exemption known as a conditional marketing authorization ,approval for Translarna (ataluren) for Duchenne Muscular Dystrophy. A conditional approval is intended to allow for medicines that may combat life-threatening illnesses when there are no other drug options available.