In a landmark decision, today the FDA approved Ryoncil from Mesoblast for GvHD. This is the first time the FDA has approved a mescenchymal stromal/stem cell or MSC therapy of any kind.
There have been many ups and downs for Mesoblast toward this approval of Ryoncil or remestemcel-L, an allogeneic marrow MSC product. See my 2023 post for more background on remestemcel-L and data expectations.
Congrats to the company.
What led to Ryoncil approval?
Some felt the FDA was likely to ask Mesoblast to produce more clinical trial data prior to any approval. However, I noted recent trends toward the FDA’s biologics branch, CBER, clearing INDs in the cell and tissue biologics space and even approving therapies with less data.
Based on these trends and expectations for the new administration, just yesterday I predicted the FDA was likely to approve Ryoncil in 2025. This was one of my 25 predictions for the stem cell and regenerative medicine space. Good timing, huh?
It seemed that many investors were betting that Mesoblast would soon get good news too. The stock has been way up recently. I wish I was a biotech stock investor, but I’m not. I do not have any direct investments in stem cell biotechs including Mesoblast.
One of the broader challenges with MSC or mesenchymal cell therapies is that the products can be so heterogeneous. Some firms stick with that heterogeneity, while others purify more uniform subpopulations.
From the FDA on the approval
In the agency press release, the FDA began:
“Today, the U.S. Food and Drug Administration approved Ryoncil (remestemcel-L-rknd), an allogeneic (donor) bone marrow-derived mesenchymal stromal cell (MSC) therapy indicated for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients 2 months of age and older.”
This approval is thus for one particular type of pediatric GvHD.
The PR continues (emphasis was from the FDA):
“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,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “This first mesenchymal stromal cell therapy approval demonstrates the FDA’s commitment to supporting the development of safe and effective products that could improve the quality of life for patients with symptoms that are unresponsive to other therapies.”
These are very striking words from Dr. Marks. They could suggest further openness to approval of other MSC therapies in the future. There are already a wide variety of MSC INDs.
Looking ahead: the FDA and mesenchymal cells, the unproven clinic problem
There’s a probable downside having nothing to do with Mesoblast.
Some of the loads of unproven U.S. stem cell clinics selling MSC-type injections are going to use the Ryoncil approval as a tool. Their goal will be to lower FDA standards and reduce oversight activity in the direct to consumer clinic space. They might succeed in some cases. Even if the supposed MSCs from the clinics are barely recognizable as cellular relatives of Mesoblast’s MSCs.
Overall, while the argument could have been made for more data prior to approval, I see this FDA approval as a likely positive move.
They will still need to prove efficacy, and that’s a steep order for MSCs. How would MSCs help autoimmune disease? I don’t see it.
I think the general idea is immuno-modulation/suppression but unclear.
Excellent news from the FDA. This is indeed only the beginning. MSC approvals for autoimmune diseases are next.