December 2020 (Est)
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Catalyst Info & Data Links
TITLE: RYONCIL™ (Remestemcel-L) in Acute graft-versus-host disease (aGVHD) – Pediatric BLA Approval
ClinicalTrial.gov (NCT02336230): A Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD
WHAT IS THE CATALYST EVENT?
WHEN WILL THE EVENT (OR DID THE EVENT) OCCUR?
2020: A phase 3 randomized study of remestemcel-L versus placebo added to second-line therapy in patients with steroid-refractory acute graft-versus-host disease. Biology of Blood and Marrow Transplantation, 26(5), 835-844.
MECHANISM OF ACTION / RATIONALE
Mesoblast’s lead allogeneic cell therapy product candidate, remestemcel-L, is an investigational therapy comprising culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. Remestemcel-L is thought to have immunomodulatory properties to counteract the cytokine storms that are implicated in various inflammatory conditions by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.
Acute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT).
Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, and these numbers are increasing.2 In patients with the most severe form of acute GVHD (Grade C/D or III/IV) mortality is as high as 90% despite optimal institutional standard of care.
Updated by MV
#MESO, #RYONCIL, #Remestemcel-L, #Acute graft-versus-host disease, #pediatric
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