FDA Approves First Mesenchymal Stromal Cell Therapy to Treat Steroid-refractory Acute Graft-versus-host Disease
The FDA has approved Ryoncil (remestemcel-L), the first mesenchymal stromal cell (MSC) therapy for treating steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients aged two months and older. This approval, announced on December 18, 2024, marks a significant milestone in cell-based therapies for severe conditions like GVHD, which can have devastating effects on patients, particularly children[1][2][4]. Ryoncil is derived from healthy donor bone marrow and has demonstrated a 70% overall response rate in clinical trials[3][5].
Citations:
[1] https://www.biopharma-reporter.com/Article/2024/12/19/fda-approves-first-mesenchymal-stromal-cell-therapy/
[2] https://www.globenewswire.com/news-release/2024/12/19/2999457/0/en/mesoblast-s-ryoncil-is-the-first-u-s-fda-approved-mesenchymal-stromal-cell-msc-therapy.html
[3] https://www.cgtlive.com/view/fda-approves-mesoblast-remestemcel-steroid-refractory-gvhd
[4] https://www.biospace.com/fda/mesoblast-wins-first-mesenchymal-stromal-cell-therapy-fda-approval-for-gvhd
[5] https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-remestemcel-l-rknd-steroid-refractory-acute-graft-versus-host-disease-pediatric
[6] https://www.oncnursingnews.com/view/fda-approves-first-msc-therapy-in-steroid-refractory-gvhd
[7] https://www.fiercepharma.com/pharma/fda-nod-gvhd-cell-therapy-gives-australian-biotech-mesoblast
[8] https://www.medscape.com/viewarticle/fda-oks-first-mesenchymal-stromal-cell-therapy-gvhd-2024a1000onu
[9] https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-mesoblasts-cell-therapy-graft-versus-host-disease-2024-12-18/
[10] https://www.nature.com/articles/s41423-023-01022-z
https://www.globenewswire.com/news-release/2024/11/07/2976830/28124/en/Graft-Versus-Host-Disease-GvHD-Treatment-Industry-Forecast-Report-2024-2032- Global-Market-Size-Forecast-to-Double-with-Emerging-Markets-Offering-Substantial-Growth-Potential.html
[12] https://www.fda.gov/news-events/press-announcements/fda-approves-first-mesenchymal-stromal-cell-therapy-treat-steroid-refractory-acute-graft-versus-host