The US Food and Drug Administration (FDA) has approved remestemcel-L (Ryoncil) as a treatment for pediatric patients 2 months of age and older with steroid-refractory acute graft-vs-host disease (SR-aGVHD). This decision is based on data from the single-arm, phase 3 MSB-GVHD001 trial, which found that 70% of patients experienced an overall response to remestemcel-L at day 28.
Patients treated with remestemcel-L had a complete response rate of 30% and partial response rate of 41%. The median duration of response was 54 days. Common adverse effects reported in at least 20% of patients included viral infections, bacterial infections, and hypertension.
Remestemcel-L has been under review since 2020, with the FDA initially requesting additional data. However, the agency accepted a resubmitted application in March 2023, which addressed issues related to chemistry, manufacturing, and control. The FDA approved this updated application after reviewing clinical data from MSB-GVHD001, which showed that remestemcel-L was effective for treating SR-aGVHD.
In the MSB-GVHD001 trial, patients received remestemcel-L at 2 x 10^6 MSCs/kg twice per week for 4 consecutive weeks. The treatment resulted in an overall response rate of 70%, exceeding a prespecified target of 45%. This trial’s findings support the approval of remestemcel-L as a treatment for pediatric patients with SR-aGVHD.
Source: https://www.onclive.com/view