The FDA has agreed to a rolling review towards the first component of a rolling submission for a Biologics License Application (BLA) for remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease (aGVHD).
The application enables individual components of the review to be submitted and evaluated on an ongoing basis rather than waiting for all sections to be completed. This approach will provide an opportunity for open-ended communication, and allow ample time for any matters raised by the FDA to be adequately addressed. Under this designation, a priority review can be requested; following the completion of the BLA.
The Phase III trial of 55 children (six months - 17 years) with aGVHD - 89% of that had Grade C/D disease - treatment with remestemcel-L resulted in a six-month survival of 69%.
The trial showed an Overall Response (OR) at day 28, which occurred in 69% of patients, predicted the highest survival at Day 100 and Day 180, which was 85% and 79%, respectively. The phase III trial met its primary endpoint of increased Day 28 OR compared with a protocol-defined historical control rate of 45% ( P = 0.0003). This data was found to be consistent with previous results where remestemcel-L was used as salvage therapy after failure of steroids and other agents.