Allogeneic hematopoietic stem cell transplantation (allo HSCT) has become a standard treatment option for a variety of hematologic malignancies, many immunodeficiency states, metabolic disorders, and hematopoietic disorders. Unfortunately, acute graft-versus-host disease (aGvHD) affects approximately 30% to 50% of patients following allo HSCT.[i] The first-line treatment for aGVHD is systemic corticosteroids which is considered standard of care. However, steroids are ineffective in 30 to 50% of these cases, resulting in poor prognosis.[ii]
On December 18, 2024, the FDA approved Ryoncil (Remestemcel-L-rknd) as the first and only off-the-shelf allogeneic bone marrow-derived mesenchymal stromal cell therapy for children who are two months old to adolescents with steroid-refractory acute graft-versus-host disease (SR-aGVHD). The recommended dosage for Ryoncil is 2.0 × 106 MSC/kg body weight per intravenous infusion given twice per week for four consecutive weeks, for a total of eight (8) infusions. Infusions should be administered at least three days apart. Additional doses may be considered based on response. [iii]
Mesoblast announced on February 11, 2026, that results from their Emergency Investigational New Drug (EIND) program had high survival rates with Ryoncil and emphasized the importance of earlier use in both children and adults with SR-aGVHD. These results offer strong support and rationale for their planned pivotal Phase III trial of early use of Ryoncil® as part of the second-line treatment regimen in adults with severe SR-aGvHD. The trial is expected to commence enrollment in the first quarter after protocol receives approval by the trial's central Institutional Review Board (IRB). If successful, the trial will support label extension of Ryoncil® for use in adults, a population approximately three times the size of the pediatric SR-aGvHD population.” [iv]
The wholesale acquisition cost for Ryoncil for 8 infusions is estimated at $1,550,000.
Article by Kathy Clark, RN, BSN, CMCN, RIT, Vice President, Director of Managed Care. For more information about how the impact on your plan, please contact your Summit ReSources care specialist. The following sources were used as reference material for this article:
[i] Di Francesco A, Raiola AM, Dominietto A, Di Grazia C, Gualandi F, Van Lint MT, Bregante S, Chiusolo P, Laurenti L, Sora F, Giammarco S, Metafuni E, Fresa A, Sica S, Angelucci E, Bacigalupo A. Acute graft versus host disease 1976-2020: reduced incidence and predictive factors. Front Med (Lausanne). 2024 Jan 24;10:1320692. doi: 10.3389/fmed.2023.1320692. PMID: 38327272; PMCID: PMC10847269.
[ii] Kovalenko I, Saleem T, Shah M, Seyedroudbari S, Golubykh K, Ali R, Mirza T, Laek B, Wahab A, Chattaraj A, Proskuriakova E, Garg C, Khan R. Novel Pharmacological Treatment Options of Steroid-Refractory Graft-versus-Host Disease. Adv Hematol. 2023 Dec 6;2023:9949961. doi: 10.1155/2023/9949961. PMID: 38094101; PMCID: PMC10718798.
[iii] Prescribing Information. Package Insert - RYONCIL
[iv] Mesoblast Press Release. https://investorsmedia.mesoblast.com/static-files/4961cd5e-9ee2-42b8-af62-a60beb61d553