PTC Therapeutics has shared that it has resubmitted its New Drug Application (NDA) to the FDA for review and potential approval of ataluren (Translarna). Ataluren is an oral small molecule treatment that allows for stop-codon read through to produce dystrophin in patients with nonsense mutations or ‘periods in the middle of their genetic sentence.’ Up to 15% of Duchenne diagnoses are caused by this type of change in the dystrophin gene.

PTC was the first company to conduct a large-scale placebo-controlled study in Duchenne. They have conducted a number of studies over the years and included a broad population of both very young children and adults. In the U.S., there are approximately 130 individuals with Duchenne currently on ataluren. Some of these individuals have been on ataluren for more than 10 years. 

In March 2024, PTC Therapeutics announced that based on feedback from the FDA, the company planned to resubmit its NDA for Translarna later in the year. The NDA submission is a drug sponsor’s request to the FDA for approval to sell and market a new drug in the U.S. During this process, the agency reviews the submitted information to determine whether the drug is safe and effective; whether the drug’s benefits outweigh its risks; whether the proposed drug label (package insert) is appropriate; and whether the drug manufacturing standards are adequate.

Following the resubmission, PPMD sent a request to the FDA in support of the review and hopeful positive outcome for ataluren. We are pleased the FDA has now received the NDA and look forward to FDA’s confirmation of their willingness for a full review and decision.  It is the only way for U.S. families to have the ability to understand the safety and efficacy of any product and provides families and their physicians the ability to make decisions.

Read PPMD’s Letter to FDA here.