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# FDA Rejects PTC Therapeutics’ NDA for Ataluren
- URL: https://www.fdaweb.com/fda-rejects-ptc-therapeutics-nda-for-ataluren/
- Published: 2017-10-25T12:00:00.000Z
- Updated: 2026-09-14T22:58:05.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139964

FDA has issued PTC Therapeutics a complete response letter for its NDA for ataluren for treating nonsense mutation dystrophinopathies. The agency letter “indicated that evidence of effectiveness from an additional adequate and well-controlled clinical trial(s) will be necessary at a minimum to provide substantial evidence of effectiveness,” the company says. The letter also mentioned other nonclinical and chemistry, manufacturing and controls issues that the company says it is addressing.

FDA’s decision came as no surprise. Last month, an agency advisory committee voted 10-1 against recommending approval. FDA medical reviewers said that the studies submitted by PTC Therapeutics did not persuasively demonstrate the drug’s effectiveness. The reviewers’ [briefing document](https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/PeripheralandCentralNervousSystemDrugsAdvisoryCommittee/UCM577349.pdf?ref=fdaweb.com) distributed in advance of the meeting said the application “contains a large number of exploratory analyses that lack interpretability and are often entirely based on unblinded data. The presentation of the data in the application is often unclear as to which analyses were used by the applicant. Ultimately, no positive results from any prospectively planned analyses that are persuasive have been provided with this application.”

Ataluren is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The therapy received marketing authorization in the European Union 8/2014 and is now available in over 25 countries.