MedPath

Remestemcel-L

Generic Name
Remestemcel-L
Brand Names
-
Drug Type
Biotech
Chemical Formula
-
CAS Number
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Unique Ingredient Identifier
H57D26Z9YK
Background

Remestemcel-L is a third-party, off-the-shelf suspension of ex-vivo cultured adult human mesenchymal stem cells intended for intravenous infusion. The mesenchymal stem cells are derived from the bone marrow of unrelated and human leukocyte antigen (HLA)–unmatched healthy adult donors and have the ability to differentiate into different tissue cells. It was approved for use in Canada in May 2012 as Prochymal for the management of refractory acute Graft versus Host Disease (aGvHD) in children who are unresponsive to systemic steroid therapies, with the approval conditional upon further trials being conducted.

aGvHD is a T-cell mediated disease that occurs after allogeneic hematopoietic stem cell transplant and is a reaction of donor immune cells against host tissues developed by 30-80% of the recipients . aGvHD is often characterized by the presence of inflammatory cascades through signalling of activated T cells. While systemic corticosteroids and other immunosuppressive agents are commonly used as first-line treatments to manage aGvHD, about 30-50% of patients with aGvHD experience inadequately control of disease with first-line therapies, putting them in a risk for poor outcomes and creating a significant clinical challenge . It is estimated that the patients with the most severe forms of refractory aGvHD that do not respond to steroid therapy have expected one-year survival rates of only 5% to 30% . Human mesenchymal stem cells (hMSCs) serve to down-regulate inflammatory responses and produce anti-inflammatory cytokines and growth factors to promote tissue repair . In clinical studies, patients treated with remestemcel-L demonstrated an improvement in their aGvHD and improved survival rates at subsequent days following intravenous infusion . Based on its tolerability and safety profile, remestemcel-L is a promising alternative to second-line immunosuppressive agents .

Indication

Indicated for in the management of acute Graft versus Host Disease (aGvHD) in pediatric patients. Acute GvHD should be refractory to treatment with systemic corticosteroid therapy and/or other immunosuppressive agents. Remestemcel-L may be used for Grades C and D of the disease in any organ. Remestemcel-L may also be used in the management of Grade B aGvHD involving any visceral organ, including the GI tract and the liver, but excluding skin.

Associated Conditions
Acute, refractory Graft Versus Host Disease
Associated Therapies
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Third time lucky for Mesoblast

Mesoblast's Ryoncil, a mesenchymal stem cell therapy for paediatric steroid-refractory acute GvHD, received FDA approval after a four-year journey. Approved based on a phase 3 trial showing 70% ORR and 30% complete response rate at 28 days, it addresses a critical unmet need. Mesoblast plans a trial in adults post-pediatric approval, despite financial constraints.
manilatimes.net
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Mesoblast's RYONCIL® is the First U.S. FDA-Approved Mesenchymal Stromal Cell (MSC) Therapy

RYONCIL (remestemcel-L) is the first FDA-approved mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older. In a Phase 3 trial, 70% of children with SR-aGvHD achieved an overall response by Day 28, indicating potential survival benefits. RYONCIL's immunomodulatory effects also suggest its use for other inflammatory diseases.
drugs.com
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FDA Approves Ryoncil (remestemcel-L-rknd) Mesenchymal Stromal Cell Therapy for Steroid-Refractory Acute Graft Versus Host Disease

FDA approves Ryoncil (remestemcel-L), the first mesenchymal stromal cell therapy for steroid-refractory acute graft versus host disease in children 2 months and older. Ryoncil demonstrated a 70% overall response rate in a Phase 3 trial, offering a life-saving treatment for children with no other options.
cgtlive.com
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FDA Approves Mesoblast's Remestemcel-L for Steroid-Refractory GvHD

The FDA approved Mesoblast's remestemcel-L, the first allogeneic MSC therapy, for steroid-refractory acute GvHD in children. Ryoncil, marketed under this approval, marks a milestone in cell-based therapies for life-threatening diseases. The decision was based on sufficient data from a phase 3 trial, showing 30% complete and 41% partial responses in treated children. Common adverse reactions included infections and fever, with precautions for hypersensitivity and infusion reactions.
onclive.com
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FDA Approves Remestemcel-L for Pediatric Steroid-Refractory Acute Graft-vs-Host Disease

The FDA approved remestemcel-L (Ryoncil) for pediatric steroid-refractory acute graft-vs-host disease (SR-aGVHD), supported by a 70% overall response rate in a phase 3 trial. The approval process faced multiple CRLs requiring additional data, culminating in a third BLA acceptance in 2024.
menafn.com
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Mesoblast's RYONCIL® Is The First U.S. FDA-Approved Mesenchymal Stromal Cell (MSC) Therapy

RYONCIL (remestemcel-L) is the first FDA-approved mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease (SR-aGvHD) in children aged 2 months and older. In a Phase 3 trial, 70% of patients achieved an overall response by Day 28, indicating potential survival benefits. RYONCIL's immunomodulatory effects suggest its use in other inflammatory diseases.

FDA Grants Revascor® (Rexlemestrocel-L) Regenerative Medicine Advanced Therapy (RMAT) Designation in Children with Congenital Heart Disease

Mesoblast Limited's Revascor® (rexlemestrocel-L) received FDA's RMAT designation for treating hypoplastic left heart syndrome (HLHS) in children, following positive trial results. The therapy aims to improve left ventricle growth, crucial for surgical success. Mesoblast plans to discuss approval pathways with FDA, highlighting the therapy's potential to address unmet medical needs in HLHS.
sharecafe.com.au
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Countdown to approval: Mesoblast readies for Ryoncil's FDA milestone

Mesoblast reported Q1 FY2025 milestones, including an expected FDA decision on Ryoncil for SR-aGvHD in children by 7 Jan 2025. The company secured manufacturing and distribution channels, completed a Pre-License Inspection, and arranged US$50m financing post-approval. Revascor achieved Rare Pediatric Disease and Orphan-Drug Designation, and Phase 3 trials for chronic low back pain are ongoing. Q1 net operating cash outflows were US$10.5m, a 26% reduction, with a cash balance of US$51.1m and US$60m in financing facilities pending approval.
quantisnow.com
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Appendix 4C Quarterly Activity Report for Quarter Ended September 30, 2024

Mesoblast Limited provided highlights for Q1 FY2025, including the resubmission of its Biologics License Application (BLA) for Ryoncil® (remestemcel-L) to treat steroid-refractory acute graft versus host disease (SR-aGvHD) in children, anticipating an FDA decision by January 7, 2025. The company secured strategic financing and maintained cost control, reducing net operating spend by 26%. Mesoblast also highlighted progress with Revascor® (rexlemestrocel-L) for pediatric congenital heart disease and chronic heart failure, and the Phase 3 program for chronic low back pain associated with degenerative disc disease. Financial management focused on ensuring capital for commercial launch while reducing cash expenditure.
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