PTC To Appeal Translarna's Complete Response Letter From US FDA

PTC Therapeutics appears to be hoping that CDER Director Woodcock will do for ataluren what she did for Sarepta's exon-skipping Duchenne muscular dystrophy treatment Exondys 51 since dispute resolution request would first go to Office of New Drugs, where Woodcock is acting director.

Rejected

PTC Therapeutics Inc.'s plans to appeal a US FDA complete response letter for Translarna (ataluren) could once again make Center for Drug Evaluation and Research (CDER) Director Janet Woodcock the decision-maker on a Duchenne muscular dystrophy drug that the agency's own clinical reviewers have concluded lacks substantial evidence of efficacy.

On Oct

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