PTC Therapeutics, Inc. has announced that based on recent feedback from the FDA, the company plans to re-submit a New Drug Application (NDA) for Translarna™ (ataluren), a dystrophin restoration therapy via stop-codon readthrough for the treatment of nonsense mutation Duchenne (nmDMD). PTC expects to make the NDA resubmission by mid-2024.

PPMD previously held a webinar with PTC to discuss the company’s activities to bring ataluren to U.S. patients and sent a request to the FDA urging them to accept PTC’s NDA and conduct a full review of ataluren. We believe that regardless of the outcome, the FDA’s review is critical for allowing families to make informed decisions about potential treatment options. We appreciate FDA’s willingness to review the resubmission and  remain optimistic about the possibility of an approved therapy that specially targets nmDMD.

Read PTC’s press release here.