• RYONCIL (remestemcel-L) is the first MSC product approved by FDA for any indication.
• RYONCIL is the first FDA-approved therapy for children aged 2 months and older, includingadolescents and teenagers, with steroid-refractory acute graft versus host disease (SRaGvHD), a life-threatening condition with high mortality rates.
• In a single-arm, multi-center, Phase 3 trial of children with SR-aGvHD, 89% of whom hadhigh severity Grade C or Grade D disease, 70% achieved an overall response by Day 28 oftreatment with RYONCIL, a measure that predicts survival in aGVHD.
• RYONCIL’S immunomodulatory effects, including inhibition of T cell activation andsecretion of pro-inflammatory cytokines, position the therapy for potential otherindications in diseases with excessive inflammation.
Melbourne, Australia; December 19 and New York, USA; December 18, 2024: MesoblastLimited (ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatorydiseases, today announced the Food and Drug Administration (FDA) approved Ryoncil®(remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the United States.RYONCIL is the only MSC therapy approved in the U.S. for any indication, and the only approvedtherapy for steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 monthsand older, including adolescents and teenagers.
Transplant physician Dr Joanne Kurtzberg, the Jerome Harris Distinguished Professor of Pediatricsand Professor of Pathology, and Director, Marcus Center for Cellular Cures at Duke UniversityMedical Center (DUMC), said: “Steroid-refractory acute graft versus host disease is a devastatingcondition with an extremely poor prognosis. From today we are able to offer RYONCIL, the firstFDA-approved treatment which will be life saving for so many children and will have a greatimpact on their families.”Annually in the United States approximately 10,000 patients undergo an allogeneic bone marrowtransplant, 1,500 of whom are children. Approximately 50% develop aGvHD and almost half ofthose do not respond to steroids, the recognized first-line treatment.1-5In a single-arm multicenter Phase 3 trial of children with SR-aGvHD, 89% of whom had high severity Grade C or GradeD disease, 70% achieved an overall response by Day 28 of treatment with RYONCIL, a measurethat predicts survival in aGVHD. RYONCIL treatment was not discontinued or interrupted in anypatient for any laboratory abnormality, and the full course was completed without interruption inmore than 85% of patients. The full Phase 3 clinical study results are available in Biology of Bloodand Marrow Transplantation.6
“We are very pleased that the FDA has granted approval of RYONCIL® and are proud of thecompany’s commitment to the GVHD community in bringing this important new treatment tochildren and families with no other acceptable options,” said Dr. Silviu Itescu, Chief Executive ofMesoblast. “With RYONCIL approval by FDA, Mesoblast has demonstrated the ability to bring thefirst MSC product to market. We will continue to work closely with FDA to obtain approval of ourother late-stage products, including REVASCOR® for cardiovascular diseases and rexlemestrocel-Lfor inflammatory pain indications, as well as expanding the indications for RYONCIL in bothchildren and adults with inflammatory conditions.”
RYONCIL will be available in the United States at transplant centers and other treating hospitals.
What is RYONCIL (remestemcel-L)
RYONCIL is an allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy indicatedfor the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatricpatients 2 months of age and older.
The recommended dosage of RYONCIL is 2 × 106 MSC /kg body weight per intravenous infusiongiven twice per week for 4 consecutive weeks. Response is assessed 28 ± 2 days after the firstdose and further treatment administered as appropriate.
About Mesoblast
Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellularmedicines for the treatment of severe and life-threatening inflammatory conditions. The therapiesfrom the Company’s proprietary mesenchymal lineage cell therapy technology platform respond tosevere inflammation by releasing anti-inflammatory factors that counter and modulate multipleeffector arms of the immune system, resulting in significant reduction of the damaginginflammatory process.
Mesoblast’s RYONCIL® (remestemcel-L) for the treatment of steroid-refractory acute graft versushost disease (SR-aGvHD) in children 2 months and older is the first FDA-approved mesenchymalstromal cell (MSC) therapy.
Mesoblast is committed to developing additional cell therapies for distinct indications based on itsremestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. RYONCIL isbeing developed for additional inflammatory diseases including SR-aGvHD in adults and biologicresistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure andchronic low back pain. The Company has established commercial partnerships in Japan, Europeand China.
References / Footnotes
1. Rashidi A et al. Outcomes and predictors of response in steroid-refractory acute graft-versushostdisease: single-center results from a cohort of 203 patients. Biol Blood Bone MarrowTransplant 2019; 25(11):2297-2302.
2. Berger M, Pessolano R, Carraro F, Saglio F, Vassallo E, Fagioli F. Steroid-refractory acute graftversus-host disease graded III-IV in pediatric patients. A mono-institutional experience with along-term follow-up. Pediatric Transplantation. 2020; 24(7):e13806
3. Biavasco F, Ihorst G, Wasch R, Wehr C, Bertz H, Finke J, Zeiser R. Therapy response ofglucocorticoid-refractory acute GVHD of the lower intestinal tract. Bone MarrowTransplantation. 2022
4. Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activityworldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and MarrowTransplantation Group including the global survey.
5. HRSA Transplant Activity Report, CIBMTR, 2019
6. Kurtzberg, J. et al. A Phase 3, Single-Arm, Prospective Study of Remestemcel-L, Ex VivoCulture-Expanded Adult Human Mesenchymal Stromal Cells for the Treatment of PediatricPatients Who Failed to Respond to Steroid Treatment for Acute Graft-versus-Host Disease. BiolBlood Marrow Transplant 26 (2020) 845-854 https://doi.org/10.1016/j.bbmt.2020.01.018