FDA Approves Mesoblast’s Remestemcel-L for Steroid-Refractory GvHD

This is a developing story and will be updated with new information as it becomes available.

The FDA has approved Mesoblast’s allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy remestemcel-L for the treatment of steroid refractory acute graft versus host disease (GvHD) in children aged 2 months or older.¹

Notably, the product is the first MSC therapy to have been approved by the FDA. It will be marketed under the name Ryoncil.

“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,” Peter Marks, MD, PhD, the director of the FDA’s Center for Biologics Evaluation and Research (CBER), said in a statement.¹ “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.”

The FDA's decision was made with regard to a biologics license application (BLA) that Mesoblast resubmitted on July 8, 2024, after 2 prior BLA submissions.^2,3 In March 2024, the FDA informed Mesoblast that the available data from the phase 3 MSB-GVHD001 clinical trial (NCT02336230), including new requested Chemistry, Manufacturing, and Control (CMC) information previously requested, were sufficient for a BLA resubmission. The new data provided further evidence from potency assays demonstrating a suitable link between product used for the pediatric indication to the adult indication.⁴

The FDA notes that 54 children were treated with remestemcel-L in the MSB-GVHD001 study after receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT).¹ Among those treated, 16 patients (30%) achieved a complete response at 28 days posttreatment and 22 patients (41%) achieved a partial response. A partial response was defined as an improved condition in 1 organ, but no change in another organ.

“Steroid-refractory acute graft-versus-host disease can have significant, wide-ranging health consequences, including damage to multiple organs, reduced quality of life and risk of death in affected patients,” Nicole Verdun, MD, the director of the Office of Therapeutic Products at CBER, added to the statement.¹ “The FDA remains dedicated to helping address the urgent unmet needs of individuals with debilitating and deadly diseases, and today’s approval is an important step in that effort.”

With regard to safety, the FDA notes that the most common adverse reactions in the trial constituted infections, fever, hemorrhage, edema, abdominal pain, and hypertension. Patients receiving remestemcel-L should receive pretreatment with corticosteroids and antihistamines and monitored for hypersensitivity reactions, dyspnea, hypotension, fever, tachypnea, cyanosis, and hypoxia, according to the agency. Furthermore, the FDA notes that acute infusion reactions, transmission of infectious disease or agents, and ectopic tissue formation may occur in patients who have been treated with the therapy.

REFERENCES

1. FDA Approves First Mesenchymal Stromal Cell Therapy to Treat Steroid-refractory Acute Graft-versus-host Disease. News release. FDA. December 18, 2024. Accessed December 18, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-mesenchymal-stromal-cell-therapy-treat-steroid-refractory-acute-graft-versus-host
2. FDA accepts Mesoblast’s biologics license application (BLA) for Ryoncil® in children with steroid-refractory acute graft-versus host disease (SR-aGVHD). https://investorsmedia.mesoblast.com/static-files/9a696437-abbb-4183-822f-9f74c1da5504
3. United States Food & Drug Administration (FDA) notifies mesoblast that available clinical data from phase 3 trial appear sufficient to support BLA submission for remestemcel-L in children with steroid refractory acute graft versus host disease (SR-aGVHD). News release. Mesoblast Limited. March 25, 2024. Accessed March 26, 2024. https://investorsmedia.mesoblast.com/static-files/cbb18b5c-3dea-42bf-877d-599dcc242c44
4. Mesoblast receives complete response from U.S. Food and Drug
Administration for biologics license application for steroidrefractory acute graft versus host disease in children. News release. Mesoblast Limited. Accessed August 4, 2023. https://investorsmedia.mesoblast.com/static-files/422cd6da-a0b9-49cf-a177-7fd106f111f2