Danielle Brill Bongero's questions to PTC Therapeutics Inc (PTCT) leadership • Q3 2024
Question
Danielle Brill Bongero of Raymond James sought clarification on why the Translarna NDA filing is considered a resubmission rather than a new NDA, despite including new data from Study 041.
Answer
CEO Matthew Klein explained the procedural history: the original NDA was filed in 2016 and received a Complete Response Letter (CRL) that mandated an additional trial. Because Study 041 was conducted specifically to address the deficiency cited in that CRL, the current filing is technically a resubmission of the original application.