The FDA’s Office of New Drugs has denied PTC Therapeutics' appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren. The FDA has recommended a possible path forward for ataluren review in the US. This would involve PTC resubmitting an NDA for ataluren under the accelerated approval framework utilizing the current efficacy and safety data in conjunction with new data to be generated on dystrophin production in nonsense mutation Duchenne patients. PTC Therapeutics is in discussions with the FDA on the methods to collect the dystrophin data and expedite this potential path forward.
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