FDA indicates way forward for ataluren application to treat Duchenne muscular dystrophy.- Translarna.

PTC Therapeutics announced that the Office of New Drugs of the FDA has reiterated the FDA's prior position and denied PTC's appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for PTC Therapeutics announced that the Office of New Drugs of the FDA has reiterated the FDA's prior position and denied PTC's appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren. In its letter, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway.

This would involve a re-submission of an NDA containing the current data on effectiveness of ataluren with new data to be generated on dystrophin production in nonsense mutation Duchenne muscular dystrophy (nmDMD) patients' muscles, as quantified by procedures to be agreed upon between PTC and the FDA and using newer technologies. The letter adds that PTC's Study 041, which is currently enrolling, could serve as the confirmatory post-approval trial required in connection with the accelerated approval framework. In a clarification teleconference with the FDA promptly after receiving the letter, PTC indicated its intent to follow the FDA's recommendation and preliminarily discussed methods to collect such dystrophin data and expedite this potential path forward. Based on these interactions, PTC currently intends to maintain patients in the U.S. currently receiving ataluren for nmDMD through an expanded access clinical program during this process.

In its letter, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway. This would involve a re-submission of an NDA containing the current data on effectiveness of ataluren with new data to be generated on dystrophin production in nonsense mutation Duchenne muscular dystrophy (nmDMD) patients' muscles, as quantified by procedures to be agreed upon between PTC and the FDA and using newer technologies.

The letter adds that PTC's Study 041, which is currently enrolling, could serve as the confirmatory post-approval trial required in connection with the accelerated approval framework. In a clarification teleconference with the FDA promptly after receiving the letter, PTC indicated its intent to follow the FDA's recommendation and preliminarily discussed methods to collect such dystrophin data and expedite this potential path forward. Based on these interactions, PTC currently intends to maintain patients in the U.S. currently receiving ataluren for nmDMD through an expanded access clinical program during this process.