PTC Therapeutics receives FDA refuse-to-file letter for DMD therapy Translarna

PTC Therapeutics announced Tuesday that it received a refuse-to-file letter from the FDA regarding its marketing application for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD). According to the company, the agency said the application for the protein restoration therapy was not sufficiently complete to permit a substantive review. Shares in PTC fell as much as 40 percent on the news.

PTC initiated a rolling submission for Translarna with the FDA in December 2014, which was completed last month. In October last year, the drugmaker unveiled late-stage study data illustrating that the drug failed to improve the distance patients walked in a six-minute walk test (for related analysis, see ViewPoints: PTC’s failed Translarna study could turn out to be a big success).

The FDA previously issued PTC a refuse-to-file letter regarding Translarna in 2011. The therapy was granted conditional approval for the treatment of nonsense DMD by European regulators in 2014.

Last month, BioMarin Pharmaceutical received a complete response letter from the FDA concerning its submission for the investigational DMD treatment Kyndrisa (drisapersen), which came after the agency delayed a final decision on the approval of the therapy in December. Meanwhile, the FDA postponed an advisory committee meeting scheduled to discuss Sarepta Therapeutics' filing for the DMD therapy eteplirsen, later extending its deadline for issuing a final decision on the drug to May 26.

For further analysis, read ViewPoints: With first wave of DMD agents in limbo, second wave coming into focus.

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