FDA grants priority review to Ryoncil for certain children with acute GVHD
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The FDA granted priority review to the allogeneic cell therapy remestemcel-L for the treatment of children with steroid-refractory acute graft-versus-host disease, according to the agent’s manufacturer.
Approximately half of patients who undergo allogeneic bone marrow transplantation develop acute GVHD. There are no FDA-approved treatments in the United States for children aged younger than 12 years who develop steroid-refractory acute GVHD.
Remestemcel-L (Ryoncil, Mesoblast Ltd.) — comprised of culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor — is administered to patients in a series of IV infusions.
The therapy is believed to have immunomodulatory properties to counteract the inflammatory processes implicated in steroid-refractory acute GVHD, according to a Mesoblast-issued press release. These include downregulating production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.
“There is a critical need to improve survival outcomes [for] children suffering from the more advanced stages of this devastating disease,” Silviu Itescu, MBBS, FRACP, CEO of Mesoblast, said in the release. “Mesoblast is on track in its preparation for the potential launch of Ryoncil, including meeting its target inventory build and commercial team rollout.”
The FDA is expected to make a decision on approval by Sept. 30.