Mesoblast announced that it has submitted its completed Biologics License Application (BLA) to the United States Food and Drug Administration (US FDA) for Ryoncil (remestemcel-L), its lead allogeneic cell therapy for the treatment of children with steroid-refractory acute graft versus host disease (SRaGVHD). Mesoblast filed the final module of the rolling BLA submission, covering quality control and manufacturing, with the FDA on January 31. The Company has requested Priority Review of the BLA by the FDA under the product candidate’s existing Fast Track designation for SR-aGVHD. If approved, Ryoncil is expected to be launched in the US in 2020.
Ryoncil has been used in 309 children with SR-aGVHD across three separate studies. Ryoncil was used as salvage therapy in an expanded access program in 241 children with SR-aGVHD (80% Grade C/D) who failed institutional standard of care. Ryoncil was also used as first-line therapy in Mesoblast’s open-label Phase 3 trial in 55 children with SR-aGVHD, 89% of whom had Grade C/D disease. The primary endpoint of Day 28 Overall Response in those exposed to remestemcel-L was achieved in 70% and Day 100 survival was 75%. These outcomes were superior to those from a cohort of 30 pediatric patients with SR-aGVHD from the MAGIC consortium matched for inclusion criteria and disease severity (80% Grade C/D). In the MAGIC controls, Day 28 Overall Response was 43% and Day 100 survival was 57%.
About Ryoncil (remestemcel-L)
Remestemcel-L, is an investigational therapy comprising culture expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. Remestemcel-L is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in aGVHD 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
About Acute GVHD
Acute GVHD is a potentially life-threatening condition which occurs in about 50% of patients who receive an allogeneic bone marrow transplant. Over 30,000 patients worldwide receive an allogeneic bone marrow transplant, primarily during treatment for blood cancers, and 20-25% are children. Mortality at 12 months is as high as 90% in those with aGVHD and Grade C/D or III/IV disease severity, and there are no approved treatments in the US for children under 12.