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# FDA Refuse to File Letter on Duchenne Drug
- URL: https://www.fdaweb.com/fda-refuse-to-file-letter-on-duchenne-drug/
- Published: 2016-02-23T12:00:00.000Z
- Updated: 2026-09-15T02:38:17.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5134865

> FDA has sent PTC Therapeutics a Refuse to File letter on the company’s NDA for Translarna (ataluren), an oral, first-in-class, protein restoration therapy for treating nonsense mutation Duchenne muscular dystrophy (DMD). The agency’s letter said the NDA was not sufficiently complete to permit a substantive review, according to the company. PTC says it is reviewing the letter to determine the appropriate next steps.  
>  
> Primarily affecting males, DMD is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with DMD lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties. Nonsense mutations account for 13% of DMD cases, or about 2,000 patients in the U.S., according to PTC.