Remestemcel, sold under the brand name Ryoncil, is an

allogeneic Allotransplant (''allo-'' meaning "other" in Greek) is the transplantation of cells, tissues, or organs to a recipient from a genetically non-identical donor of the same species. The transplant is called an allograft, allogeneic transplant, ...

bone marrow-derived mesenchymal stromal

cell therapy Cell therapy (also called cellular therapy, cell transplantation, or cytotherapy) is a therapy in which viable cells are injected, grafted or implanted into a patient in order to effectuate a medicinal effect, for example, by transplanting T- ...

used for the treatment of

graft-versus-host disease Graft-versus-host disease (GvHD) is a syndrome, characterized by inflammation in different organs. GvHD is commonly associated with bone marrow transplants and stem cell transplants. White blood cells of the donor's immune system which rema ...

. Remestemcel contains mesenchymal stromal cells, which are a type of cell that can have various roles in the body and can differentiate into multiple other types of cells. These mesenchymal stromal cell are isolated from the bone marrow of healthy adult human donors. The most common adverse reactions include viral infectious disorders, bacterial infectious disorders, infection – pathogen unspecified, pyrexia, hemorrhage, edema, abdominal pain, and hypertension. Remestemcel was approved for medical use in the United States in December 2024. Remestemcel is the first mesenchymal stromal cell therapy approved by the US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

Medical uses

Remestemcel is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

for the treatment of steroid-refractory acute

History

The safety and effectiveness of remestemcel were evaluated in a multicenter, single-arm study in 54 pediatric study participants with steroid-refractory acute graft-versus-host disease after undergoing allogeneic hematopoietic (blood) stem cell transplantation. Study participants received intravenous infusion of remestemcel twice weekly for four consecutive weeks, for a total of eight infusions. Each study participant's condition at baseline was analyzed using the international blood and marrow transplantation registry severity index criteria (IBMTR) to evaluate which organs have been affected and the overall severity of the disease. The effectiveness of remestemcel was based primarily on the rate and duration of response to treatment 28 days after initiating remestemcel. Study participants who had a partial or mixed response to treatment—meaning that there was improved condition in one organ with either no change (partial) or worsening condition (mixed) in another organ—received additional infusions once weekly for an additional four weeks. Sixteen study participants (30%) had a complete response to treatment 28 days after receiving remestemcel, while 22 study participants (41%) had a partial response. The US

(FDA) granted the application for remestemcel

fast track Fast Track, Fast track, or Fasttrack may refer to: Processes and systems * Fast track (FDA), a U.S. Food and Drug Administration expedited review program * Fast track (trade), the authority of the U.S. President to broker trade agreements with lim ...

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

, and

priority review Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...

designations. The FDA granted approval of Ryoncil to Mesoblast, Inc.

Society and culture

Legal status

Remestemcel was approved for medical use in the United States in December 2024.

Names

Remestemcel is the international nonproprietary name. Remestemcel-L is the

United States Adopted Name A United States Adopted Name (USAN) is a unique nonproprietary name assigned to a medication marketed in the United States. Each name is assigned by the USAN Council, which is co-sponsored by the American Medical Association (AMA), the United St ...

References

External links

* * {{Authority control Medical treatments Cell therapies