Ataluren not approved by FDA for nonsense mutation muscular dystrophies

PTC Therapeutics, Inc. announced that the Office of Drug Evaluation 1 of the FDA has issued a complete response letter (CRL) for the New Drug Application (NDA) of the investigational medicine Translarna (ataluren) for the treatment of nonsense mutation dystrophinopathies. 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.