In a corporate update released today, PTC Therapeutics announced next steps for their drug, Translarna in the U.S. According to the release, PTC plans to file the Translarna New Drug Application (NDA) for nmDMD over protest with the U.S. FDA in the first quarter of 2017. PTC stated that feedback indicated this process, rather than continued appeal, is the best path forward for the current Translarna NDA to receive a full and fair review.
Filing over protest is a procedural path permitted by U.S. FDA regulations that allows a company to have its NDA filed and reviewed when there is a disagreement with regulators over the acceptability of the NDA submission. PTC plans to supplement the current NDA with additional efficacy analyses utilized by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in their recent renewal recommendation
PPMD remains hopeful that the positive data from studies to date will provide a regulatory path for Translarna’s approval.
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