Rare Daily Staff

The U.S. Food and Drug Administration told PTC Therapeutics it would not approve its experimental therapy Translarna for Duchene muscular dystrophy for the treatment of a certain form of Duchene muscular dystrophy, a rare and fatal progressive muscle disease.

The agency’s ruling followed an advisory panel 10 to 1 to recommend against approval. It was the third attempt by PTC to win approval of Translarna for DMD driven by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein, such a dystrophin in the case of DMD.

“We are extremely disappointed for the Duchenne community and strongly disagree with the agency’s conclusions,” said Stuart Peltz, CEO of PTC Therapeutics. “We believe that this decision fails to consider the benefit-risk of ataluren and the high unmet medical need. Therefore, we plan to file a formal dispute resolution request next week.”

In a complete response letter to the company, the FDA said that it is unable to approve the application in its current form. It said it would require at least one additional clinical trial to provide substantial evidence of effectiveness. The letter also noted other nonclinical matters that PTC said it is in the process of addressing.

Ataluren is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. Translarna won conditional approval in the European Union in 2014 for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older.

October 25, 2017

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