Remestemcel-l

The mechanism of action for RYONCIL is not clear but may be related to immunomodulatory effects. Data from in vitro studies demonstrate that MSCs inhibit T cell activation as measured by proliferation and secretion of pro-inflammatory cytokines.

• RYONCIL is indicated for the treatment of steroid refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older. RYONCIL is an allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy indicated for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older.

• Do not use RYONCIL in patients with known hypersensitivity to dimethyl sulfoxide (DMSO) or porcine and bovine proteins. Known hypersensitivity to dimethyl sulfoxide (DMSO) or Porcine and Bovine proteins.contraindicated

For intravenous use only. For intravenous use only. The recommended dosage of RYONCIL is 2 × 10 6 MSC/kg body weight per intravenous infusion given twice per week for 4 consecutive weeks. Infusions should be administered at least 3 days apart. Assess response 28 ± 2 days after the first dose and administer further treatment as appropriate as described in the table below. Recommended Treatment Based on Day 28 Response Response Recommendation Complete Response (CR) No further treatment with RYONCIL Partial or Mixed Response Repeat administration of RYONCIL once a week for additional 4 weeks (4 infusions total) No Response Consider alternative treatments Recurrence of GvHD after CR Repeat administration of RYONCIL twice a week for an additional 4 consecutive weeks (8 infusions total) 2.1 Recommended Dosage The recommended dosage of RYONCIL is 2 × 10 6 mesenchymal stromal cells (MSC)/kg body weight per intravenous infusion given twice a week for 4 consecutive weeks for a total of 8 infusions. Administer infusions at least 3 days apart. Assess response 28 ± 2 days after the first dose and administer further treatment as appropriate as described in Table 1 based on Day 28 response.

Hypersensitivity/Acute Infusion reactions : Monitor for hypersensitivity reactions during infusion and premedicate with corticosteroids and antihistamines. ( 5.1 ) Transmission of Infectious Agents: RYONCIL may transmit infectious agents. ( 5.2 ) Ectopic Tissue Formation: Ectopic tissue formation may occur following treatment with RYONCIL. ( 5.3 ) 5.1 Hypersensitivity and Acute Infusion Reactions Hypersensitivity reactions including acute infusion reactions have occurred with RYONCIL administration [see Adverse Reactions (6.1) ] . Serious hypersensitivity reactions, including anaphylaxis, may occur due to DMSO and trace amounts of porcine or bovine proteins. Signs and symptoms may include fever, dyspnea, and hypotension during or after RYONCIL infusion. Premedicate patients with antihistamine and corticosteroids and monitor closely for signs and symptoms of hypersensitivity or acute infusion reactions. If a hypersensitivity or infusion reaction occurs, interrupt RYONCIL infusion. Do not administer RYONCIL in patients who experience serious or life-threatening reactions. 5.2 Transmission of Infectious Agents Transmission of infectious disease or agents may occur with RYONCIL administration because it contains cells from human donors and is manufactured using human, porcine and bovine-derived reagents.

The most common non-laboratory adverse reactions (incidence ≥20%) are: viral infectious disorders, bacterial infectious disorders, infection – pathogen unspecified, pyrexia, hemorrhage, edema, abdominal pain and hypertension ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Mesoblast at toll-free phone #1-844-889-MESO (6376) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical studies are conducted under widely varying conditions, adverse reaction rates observed in the clinical studies of a drug cannot be directly compared to rates in the clinical studies of another drug and may not reflect the rates observed in practice. The safety data described in this section reflect exposure to RYONCIL in 54 patients in Study MSB-GVHD001 for the treatment of SR-aGvHD. Patients received intravenous infusion of RYONCIL at a dosage of 2 x 10 6 MSCs/kg twice a week for four consecutive weeks, for a total of eight infusions. Patients with partial or mixed response at Day 28 received additional infusions of RYONCIL 2 x 10 6 MSCs/kg once a week for an additional four weeks [see Clinical Studies (14) ] . The median number of doses administered were 10 (range 1 to 16), and the treatment was administered over a median of 43 days (range 1 to 104 days). Serious adverse reactions occurred in 35 patients (65%) including pyrexia (n=5;9%), respiratory failure (n=5;9%), pneumatosis intestinalis (n=4;7%) and staphylococcal bacteremia (n=2;<5%).

8.1 Pregnancy Risk Summary There are no available data for RYONCIL use in pregnant women. No animal reproductive and developmental toxicity studies have been conducted with RYONCIL to assess whether it can cause fetal harm when administered to a pregnant woman. It is not known if RYONCIL has the potential to be transferred to the fetus. Therefore, RYONCIL is not recommended for women who are pregnant. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 10-20%, respectively. 8.2 Lactation Risk Summary There is no information regarding the presence of RYONCIL in human milk, the effect on the breastfed infant, and the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for RYONCIL and any potential adverse effects on the breastfed infant from RYONCIL or from the underlying maternal condition. 8.4 Pediatric Use The safety and effectiveness of RYONCIL for treatment of SR-GvHD have been established in pediatric patients 2 months of age and older. The use of RYONCIL in these age groups is supported by evidence from an adequate and well-controlled trial [see Adverse Reactions (6) and Clinical Studies (14) ] .

Metabolism

No studies have been performed to evaluate the pharmacokinetics of RYONCIL.

The 10 most recently updated of 21 ClinicalTrials.gov registrations naming Remestemcel-l as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase III Trial of Remestemcel-L-rknd Added to Ruxolitinib for Grade III-IV Steroid-Refractory Acute Graft-Versus-Host Disease
  Not yet recruiting · Phase 3 · Interventional · 180 enrolled · Mesoblast, Ltd.
  NCT07568535updated 2026-05-05
• Study of Mesenchymal Stem Cells for the Treatment of Medically Refractory Crohn's Colitis
  Terminated · Phase 1 · Phase 2 · Interventional · 7 enrolled · The Cleveland Clinic
  NCT04548583updated 2026-04-16
• Study of Mesenchymal Stem Cells for the Treatment of Medically Refractory Ulcerative Colitis (UC)
  Terminated · Phase 1 · Phase 2 · Interventional · 7 enrolled · The Cleveland Clinic
  NCT04543994updated 2026-04-16
• PROCHYMAL™ (Human Adult Stem Cells) for the Treatment of Moderate to Severe Chronic Obstructive Pulmonary Disease (COPD)
  Completed · Phase 2 · Interventional · 62 enrolled · Mesoblast, Inc.
  NCT00683722updated 2026-03-11
• Prochymal® (Human Adult Stem Cells) Intravenous Infusion Following Acute Myocardial Infarction (AMI)
  Completed · Phase 2 · Interventional · 220 enrolled · Mesoblast, Inc.
  NCT00877903updated 2026-03-11
• Evaluation of PROCHYMAL® Adult Human Stem Cells for Treatment-resistant Moderate-to-severe Crohn's Disease
  Completed · Phase 3 · Interventional · 330 enrolled · Mesoblast, Inc.
  NCT00482092updated 2026-03-11
• PROCHYMAL® (Human Adult Stem Cells) for the Treatment of Recently Diagnosed Type 1 Diabetes Mellitus (T1DM)
  Completed · Phase 2 · Interventional · 63 enrolled · Mesoblast, Inc.
  NCT00690066updated 2026-03-11
• Evaluation of PROCHYMAL® for Treatment-refractory Moderate-to-severe Crohn's Disease
  Completed · Phase 3 · Interventional · 73 enrolled · Mesoblast, Inc.
  NCT01233960updated 2026-03-11
• Intermediate-size Expanded Access Program (EAP), Mesenchymal Stromal Cells (MSC) for Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)
  No longer available · Expanded access · Mesoblast International Sàrl
  NCT04456439updated 2025-10-31
• Safety and Efficacy of Prochymal for the Salvage of Treatment-Refractory Acute GVHD Participants
  Completed · Phase 2 · Interventional · 11 enrolled · Mesoblast, Inc.
  NCT00284986updated 2025-10-30

Frequently asked questions

How does Remestemcel-l work?

The mechanism of action for RYONCIL is not clear but may be related to immunomodulatory effects. Data from in vitro studies demonstrate that MSCs inhibit T cell activation as measured by proliferation and secretion of pro-inflammatory cytokines.

What is Remestemcel-l used for?

According to FDA labeling, Remestemcel-l carries indications including: RYONCIL is indicated for the treatment of steroid refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older. RYONCIL is an allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy indicated for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Remestemcel-l?

Remestemcel-l is classified as Biological Response Modifiers, Decreased Cytokine Production, Decreased Immunologic Activity.

What are the brand names for Remestemcel-l?

Remestemcel-l is marketed under brand names including Ryoncil.

What are the contraindications for Remestemcel-l?

Remestemcel-l labeling lists contraindications including: Do not use RYONCIL in patients with known hypersensitivity to dimethyl sulfoxide (DMSO) or porcine and bovine proteins. Known hypersensitivity to dimethyl sulfoxide (DMSO) or Porcine and Bovine proteins.. Always consult the full prescribing information and a clinician.