from PTC Therapeutics
SOUTH PLAINFIELD, N.J., Oct. 25, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Office of Drug Evaluation I of the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) for the New Drug Application (NDA) of the investigational medicine ataluren for the treatment of nonsense mutation dystrophinopathies.
“We are extremely disappointed for the Duchenne community and strongly disagree with the agency’s conclusions,” said Stuart W. Peltz, Ph.D., chief executive officer of PTC Therapeutics. “We believe that this decision fails to consider the benefit-risk of ataluren and the high unmet medical need. Therefore, we plan to file a formal dispute resolution request next week.”
The letter from the Office of Drug Evaluation I of the FDA stated that it is unable to approve the application in its current form. Specifically, the 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 letter also mentioned other nonclinical and CMC matters that PTC is in the process of addressing.
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