FDA Fails to Approve PTC Therapeutics NDA for Ataluren for Nonsense Mutation Dystrophinopathies.
M2 PHARMA-October 26, 2017-FDA Fails to Approve PTC Therapeutics NDA for Ataluren for Nonsense Mutation Dystrophinopathies
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- The Office of Drug Evaluation I of the US Food and Drug Administration has issued South Plainfield, New Jersey-based RNA biology specialist PTC Therapeutics, Inc. (NASDAQ: PTCT) a complete response letter for the company's New Drug Application of the investigational medicine ataluren for the treatment of nonsense mutation dystrophinopathies, the company said.
The letter from the FDA said that it is unable to approve the application in its current form, and 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 letter also mentioned other nonclinical and CMC matters that PTC is in the process of addressing.
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, that is, an alteration in the genetic code that prematurely halts the synthesis of an essential protein.
Translarna, trade name of ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. It is an investigational new drug in the United States.
PTC is a global biopharmaceutical company focused on the discovery, development, and commercialization of treatments for rare genetic disorders and oncology. The company has discovered all of its compounds currently under development using its proprietary technologies.
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|Date:||Oct 26, 2017|
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