Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide a quick update on a recent FDA cancer therapeutic approval.
On December 18, 2024, the FDA approved remestemcel-L-rknd (brand name Ryoncil), an allogeneic bone marrow-derived mesenchymal stromal cell therapy, for steroid-refractory acute graft versus host disease in pediatric patients 2 months of age and older. Ryoncil is the first FDA-approved mesenchymal stromal cell therapy.
Efficacy was evaluated in MSB-GVHD001, a multicenter, prospective, single-arm study in 54 pediatric patients with steroid-refractory acute graft versus host disease after allogeneic hematopoietic stem cell transplantation. Patients had Grade B to D steroid-refractory acute graft versus host disease, excluding Grade B skin alone (based on the International Blood and Marrow Transplantation Registry Severity Index Criteria). Steroid-refractory acute graft versus host disease was defined as acute graft versus host disease progressing within 3 days or not improving within 7 consecutive days of methylprednisolone. Patients receiving a second-line acute graft versus host disease therapy prior to screening were excluded.
The main efficacy outcome measures were overall response rate at Day 28 and duration of overall response. The overall response rate included complete and partial response rates. Overall response rate at Day 28 was 70%, including a complete response rate of 30% and a partial response rate of 41%. The median duration of response calculated from response at Day 28 to either progression, new systemic therapy for acute graft versus host disease, or any cause death, was 54 days.
The most common nonlaboratory adverse reactions reported in more than 20% of patients were viral infectious disorders, bacterial infectious disorders, infection – pathogen unspecified, pyrexia, hemorrhage, edema, abdominal pain and hypertension.
Full prescribing information for these approvals can be found on the web at www.fda.gov, key word search Approved Cellular and Gene Therapy Products.
Health care professionals should report serious adverse events to www.fda.gov/medwatch.
Follow the Division of Drug Information on X @FDA_Drug_Info and the Oncology Center of Excellence @FDAOncology. Send your feedback via email to FDAOncology@fda.hhs.gov.
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