FDA quells PTC Therapeutic’s appeal for NDA for ataluren

PTC Therapeutics asserted that the office of New Drugs of the U.S Food and Drug Administration has reaffirmed the FDA’s former position quashing PTC’s appeal of the Complete Response Letter in relation to the New Drug Application for ataluren.

The letter suggests the Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway. 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 suggested that its intent was to follow the FDA’s recommendation and preliminary discussed methods to collate such dystrophin data and facilitate this potential path forward.