EMA To Establish Minimum Requirements For CAR-T Cell Registries

The European Commission has adopted new rules to cut red tape in pharmacovigilance, tighten oversight of outsourced drug safety activities, and ensure a balanced approach to signal detection, boosting both efficiency and patient safety.

The FDA recommended removal of a voluntary hold on shipments of the gene therapy to younger patients after finding that the death of an 8-year-old boy in Brazil was unrelated to Elevidys. Discussions on safety of DMD treatment for non-ambulatory patients is ongoing.

One option for the FDA going forward could be holding an advisory committee meeting on its safety concerns after Sarepta agreed to stop all shipments of its DMD gene therapy.

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.