A Milestone in Regenerative Medicine: FDA Approves First MSC Therapy
- DPS
- Jul 21
- 1 min read
In a landmark decision in December 2024, the U.S. Food and Drug Administration (FDA) approved Remestemcel‑L (brand name: Ryoncil)—marking the first mesenchymal stromal cell (MSC) therapy to gain FDA approval.
This therapy is indicated for steroid‑refractory acute graft‑versus‑host disease (aGVHD) in pediatric patients. aGVHD is a life‑threatening complication that can occur after hematopoietic stem cell transplantation, where the donor’s immune cells attack the patient’s tissues. Until now, treatment options for children who did not respond to steroids were extremely limited.
Why This Approval Matters
MSC therapies, derived from bone marrow and expanded in controlled conditions, are known for their immunomodulatory and anti‑inflammatory properties. In clinical studies, Remestemcel‑L demonstrated:
Improved overall survival compared to historical outcomes in steroid‑refractory aGVHD.
A well‑tolerated safety profile, even in critically ill pediatric patients.
This approval sets a precedent for other MSC‑based therapies currently in development for conditions such as inflammatory bowel disease, cardiac injury, and autoimmune disorders.
Looking Ahead
The success of Remestemcel‑L highlights the growing maturity of cell‑based therapeutics. It not only offers new hope to children facing aGVHD but also paves the way for wider adoption of off‑the‑shelf cellular therapies in mainstream medicine.
Sources
Mesoblast. FDA Approval of Remestemcel‑L (Ryoncil) – Press Release, Dec 2024.
ASGCT News. FDA Approves First Mesenchymal Stromal Cell Therapy. Read here.
U.S. FDA. Approved Cellular and Gene Therapy Products. FDA website.
