Initial US Approval:
2024
Key Clinical Studies:
MSB-GVHD001 (NCT02336230)
Drug Class/Description:
Allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy
Indications and Usage:
Treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older.
Dosage Administration:
- The recommended dosage of RYONCIL is 2 × 106 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) |
Dosage Forms and Strengths:
RYONCIL is available as a cell suspension for intravenous infusion in a target concentration of 6.68 X 106 MSCs per mL in 3.8 mL contained in a 6 mL cryovial.
Contraindications:
Known hypersensitivity to dimethyl sulfoxide (DMSO) or Porcine and Bovine proteins.
Warnings and Precautions:
- Hypersensitivity/Acute Infusion reactions: Monitor for hypersensitivity reactions during infusion and premedicate with corticosteroids and antihistamines.
- Transmission of Infectious Agents: RYONCIL may transmit infectious agents.
- Ectopic Tissue Formation: Ectopic tissue formation may occur following treatment with RYONCIL.
Adverse Reactions:
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.
Adapted From:
https://www.ryoncil.com/pdfs/prescribing-information.pdf
Every health-care provider should make their own determination regarding specific safe and appropriate patient care practices, including drug dosages and indications. The provider should always consult the most recent prescribing/product information. FDA Focus information is not guaranteed to be accurate, complete, or current. JADPRO and its editors, authors, reviewers, and commentators cannot be held responsible for any liability incurred as a consequence of the application of any of the information listed within.
