PTC Therapeutics, Inc. announced that the Office of New Drugs of the U.S. Food and Drug Administration 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.
While incredibly disappointing, PPMD is hopeful that this possible path forward will provide an approval and continued access to the hundreds of families currently on ataluren. According to their letter to the community, PTC intends to keep supplying ataluren to patients at least until the re-submission of the NDA in the U.S. We appreciate that PTC remains dedicated to the approval of ataluren and that the FDA is open to re-examining the therapy once new data is provided. But most of all, we are grateful for all of the young men and their families participating in this trial, many who have spent over a decade on this drug. PPMD has reached out to PTC and will share any updates as they become available.