FDA Approves First Mesenchymal Stromal Cell (MSC) Therapy, a Major Milestone in Regenerative Medicine
The Food and Drug Administration (FDA) has approved RYONCIL®, the first mesenchymal stromal cell (MSC) therapy, for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in children aged two months and older.
Developed and patented by Osiris Therapeutics, a company founded by Kevin Kimberlin and Dr. Arnold Caplan, this approval marks a significant step in regenerative medicine.
Osiris Therapeutics played a pivotal role in the development of MSC therapies. The safety and potential of MSC treatments, ultimately leading to the development and FDA approval of therapies like RYONCIL.
To complete the necessary Phase 3 trials, Osiris licensed the intellectual property and sold the product, initially named Prochymal, to Mesoblast Limited, helping to further progress MSC treatments.
The approval of RYONCIL is a validation of the pioneering work done by the team at Osiris and signals a future where MSCs may offer treatments for a range of challenging medical conditions. Originally considered a stem cell, the MSC was first characterised by Osiris scientists in a paper published in Science in 1999, which has since been widely cited.
Research has shown that MSCs play a crucial role in the body's response to injury and infection, acting as a key part of the healing process.
RYONCIL joins other notable MSC innovations from Osiris, including Osteocel®, the first product in the U.S. containing adult MSCs, and Grafix®, which is now a standard treatment for chronic diabetic foot ulcers.
RYONCIL will soon be available for children at transplant centres and hospitals across the United States. Meanwhile, Mesoblast is advancing clinical trials for MSC-based treatments aimed at addressing cardiovascular conditions and lower back pain.
