Model-Informed Drug Development Program Could Handle More Requests, But US FDA Needs Funding

The European Medicines Agency has revised the sponsor handbook for the Clinical Trials Information System, bringing together guidance that was previously scattered across multiple platforms and documents to help sponsors navigate the trial process more efficiently.

Sarepta knew about the death in a Phase I trial of SRP-9004 for LGMD when it recently revealed a restructuring plan, but did not disclose the event. The now-discontinued gene therapy candidate uses the same AAV vector as Elevidys, which has been linked to two DMD patient deaths.

The UK Health Research Authority’s latest three-year strategy sets ambitious targets to make research more transparent. One World Health Organization official believes the authority can go further by setting equally high standards for the timely reporting of clinical trial results.

Rexulti’s post-traumatic stress disorder sNDA tries to make up for a failed Phase III study by placing a Phase II study into a key evidentiary role, raising questions for the July 18 advisory committee meeting about assessing discordant results and post-hoc analyses.