FDA approves remestemcel-L for the treatment of pediatric patients with SR-aGvHD

On December 18, 2024, the U.S Food and Drug Administration (FDA) approved remestemcel-L-rknd, an allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy, for the treatment of steroid-refractory acute graft-versus-host disease (aGvHD) in pediatric patients (aged ≥2 months).¹ This is the first approved MSC product in the U.S.

Approval was based on data from MSB-GVHD001 (NCT02336230), which evaluated remestemcel-L-rknd in pediatric patients with aGvHD.

MSB-GVHD001 study²

• MSB-GVHD001 was a multicenter, single-arm phase III trial of remestemcel-L-rknd in pediatric patients with aGVHD.
• In total, 54 pediatric patients with steroid-refractory aGVHD who had undergone allo-HSCT were treated with remestemcel-L-rknd.
• After 28 days of treatment, the overall response rate was 70%, and complete remission and partial remission rates were 30% and 41%, respectively. The median duration of response was 54 days.
• The most common adverse events (incidence ≥20%) were infections, fever, hemorrhage, edema, abdominal pain, and hypertension.
• The recommended dose is 2 × 10⁶ MSC/kg body weight per intravenous infusion, to be given twice a week for 4 weeks, for a total of eight infusions.² Treatment may be continued based on response to initial treatment for 28 days.