After receiving FDA feedback on its application review, PTC Therapeutics has decided to withdraw its New Drug Application (NDA) resubmission for Translarna (ataluren), an oral drug to treat Duchenne muscular dystrophy (DMD).
PTC came to the decision after the FDA shared that it was unlikely to approve the drug with the data in the NDA submission, as it does not meet the agency’s threshold of evidence of effectiveness. The FDA originally issued a Refuse to File letter in 2016 due to an incomplete application. It followed this with a Complete Response Letter (CRL) in 2017, citing the need for additional clinical trials.
"We have worked tirelessly for over two decades to develop a safe and effective therapy for boys and young men affected by nonsense mutation DMD in the U.S. and are disappointed that FDA approval cannot be achieved," said Matthew Klein, M.D., PTC Therapeutics' CEO.
Translarna is a protein restoration therapy that enables the formation of a functioning protein in patients who have a nonsense mutation, which prevents the protein from being expressed in its entirety. In DMD, the drug enables synthesis of dystrophin.