PPMD Community

PTC Provides Update on Next Steps for Translarna

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.

 

Click here to read the full release.

 

To ensure you are kept up to date about Translarna, make sure you register or update your registration in DuchenneConnect today.

Views: 354

Comment

You need to be a member of PPMD Community to add comments!

Join PPMD Community

© 2017   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service