In a significant milestone for pediatric medicine, the FDA has accepted Mesoblast’s Biologics License Application (BLA) for Ryoncil® (remestemcel-L), aimed at treating children with Steroid-Refractory Acute Graft-Versus-Host Disease (SR-aGVHD).
This acceptance marks a crucial step towards the approval of the first allogeneic “off-the-shelf” cellular medicine in the United States, and the first cell therapy for children up to 18 years old suffering from this severe condition.
Key Points:
- Ryoncil is derived from mesenchymal stromal cells from the bone marrow of unrelated donors.
- The therapy involves a series of intravenous infusions that help modulate immune responses and reduce inflammation.
- The FDA has granted Fast Track and Priority Review designations, expediting the review process.
- Results from the Phase 3 trial indicate a significant improvement in Day 28 Overall Response (OR) and Day 100 survival rates for children treated with Ryoncil compared to standard therapies.
Mesoblast CEO Dr. Silviu Itescu expressed optimism, highlighting the potential of Ryoncil to address a critical unmet need in pediatric healthcare. With the FDA’s decision expected by January 7, 2025, there is renewed hope for improved survival outcomes for children battling SR-aGVHD.
For more detailed information, visit Mesoblast.