Ryoncil is first FDA-approved mesenchymal stromal cell therapy for pediatric patients aged 2 months or older
By Elana Gotkine HealthDay Reporter
FRIDAY, Dec. 27, 2024 (HealthDay News) — The allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy Ryoncil (remestemcel-L-rknd) has been approved for treatment for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients aged 2 months or older, according to a press release from the U.S. Food and Drug Administration.
Ryoncil is the first FDA-approved MSC therapy. MSCs are isolated from the bone marrow of healthy adult human donors and are used to treat SR-aGVHD in patients who undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT).
The safety and efficacy of Ryoncil was assessed in a multicenter, single-arm study involving 54 pediatric patients who had SR-aGVHD after allo-HSCT. Participants received intravenous Ryoncil infusion twice weekly for four weeks. The effectiveness of Ryoncil was based on the rate and duration of response to treatment after 28 days of initiation. Participants with a partial or mixed response to treatment received additional infusions once weekly for four weeks. Overall, 30 percent of participants had a complete response to treatment 28 days after initiation and 41 percent had a partial response. Infections, fever, hemorrhage, edema, abdominal pain, and hypertension were the most common adverse reactions among participants receiving Ryoncil.
“Today’s decision marks an important milestone in the use of innovative cell-based therapies to treat life-threatening diseases with devastating impacts on patients, including children,” Peter Marks, M.D., Ph.D., director of the FDA Center for Biologics Evaluation and Research, said in a statement.
Approval of Ryoncil was granted to Mesoblast.
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