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# FDA Accepts PTC NDA ‘Protestֺ’ Resubmission for Duchenne Drug
- URL: https://www.fdaweb.com/fda-accepts-ptc-nda-protest-resubmission-for-duchenne-drug/
- Published: 2017-03-06T12:00:00.000Z
- Updated: 2026-09-14T22:10:07.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5138134

FDA has accepted for review a PTC Therapeutics NDA that was filed in protest for Translarna (ataluren) for treating nonsense mutation Duchenne muscular dystrophy (DMD). The move follows the company’s loss in October of an appeal after receiving a refuse-to-file letter ([see story](https://www.fdaweb.com/cder-denies-ptc-therapeutics-appeal-on-duchenne-drug/)) issued by the Division of Neurological Products. At the time, the agency said the submission was not sufficiently complete to permit a substantive review, according to the company. FDA has granted a standard review and assigned a user fee review target date of 10/24.

The protest filing is a “procedural path permitted by FDA regulations that allows a company to have its NDA filed and reviewed when there is a disagreement with regulators over the acceptability of the NDA submission,” PTC says. It reportedly supplemented the current NDA with additional efficacy analyses used by European regulators.

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.

  
Translarna received marketing authorization for patients with nmDMD in the European Union in August 2014 and is now available in over 25 countries.