PTC Therapeutics dropping after release of Briefing Docs: Memorandum for New Drug Application (NDA) 200896, for the use of Translarna (ataluren) for the treatment of dystrophinopathies resulting from nonsense mutations in the dystrophin gene
"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. In the one instance where an exploratory analysis (the unblinded post hoc analysis of Study 007) was prospectively tested (in Study 020), the results were clearly negative."
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Sept 26 (Reuters) - PTC Therapeutics Inc has not supplied persuasive evidence that its experimental drug to treat a form of Duchenne muscular dystrophy is effective, a preliminary review by scientists at the U.S. Food and Drug Administration concluded.
The review, posted on the FDA's website on Tuesday, said data to establish the effectiveness of the drug, ataluren, "are not persuasive."
The FDA's comments come two days ahead of a meeting of outside advisors who will discuss ataluren. The agency has asked its advisors to decide whether or not the drug is effective or whether the data is inconclusive.
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