FDA grants priority review of remestemcel-L for certain children with acute GvHD

On the 1st of April, the U.S. Food and Drug Administration (FDA) accepted a Biologics License Application (BLA) for remestemcel-L for the treatment of children with steroid-refractory graft-versus-host disease (SR-GvHD) and granted a Priority Review designation.¹

Remestemcel-L is an investigational therapy comprising of ex-vivo cultured adult human mesenchymal stem cells derived from the bone marrow of an unrelated healthy donor. The efficacy of remestemcel-L infusions was assessed in two clinical trials. The initial study (NCT00759018) conducted in pediatric patients with SR-GvHD demonstrated a 65% overall response (OR) at Day 28 and increased survival at Day 100 in responders compared with non-responders (82% vs 39%, p < 0.001).² A further phase III study (NCT02336230) in pediatric patients with high-risk SR-GvHD demonstrated remestemcel-L as a relatively safe and effective treatment option for pediatric patients with SR-acute GvHD that induced a durable overall response rate of 70.4% with rates for complete responses improving from Day 28 up to Day 100.³ More details on the study design and results is available here.