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# PTC Therapeutics Protest NDA Planned for Duchenne Drug
- URL: https://www.fdaweb.com/ptc-therapeutics-protest-nda-planned-for-duchenne-drug/
- Published: 2017-01-09T12:00:00.000Z
- Updated: 2026-09-14T21:58:50.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5137660

PTC Therapeutics says it plans to file in protest an NDA for Translarna (ataluren) for treating nonsense mutation Duchenne muscular dystrophy (DMD) this quarter. 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.

The protest filing is a “procedural path permitted by U.S. 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 plans to supplement 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.