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.

  1. Mesoblast. FDA accepts Mesoblast’s Biologics Licence Application for RYONCIL™ and agrees to priority review.  Published Apr 1, 2020.Accessed Apr 2, 2020.

  2. Kurtzberg J, et al. Effect of human mesenchymal stem cells (remestemcel-L) on clinical response and survival confirmed in a large cohort of pediatric patients with severe high-risk steroid-refractory acute graft versus host disease. Biol Blood Marrow Tr.2016;22(3):S59. DOI: 1016/j.bbmt.2015.11.350

  3. Kurtzberg J, Abdel-Azim H, Carpenter P, et al. A phase 3, single-arm, prospective study of remestemcel-L, ex vivo culture-expanded adult human mesenchymal stromal cells for the treatment of pediatric patients who failed to respond to steroid treatment for acute graft-versus-host disease. Biol Blood Marrow Tr. 2020 . DOI: 1016/j.bbmt.2020.01.018

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