US FDA Questions Adequacy Of Mesoblast’s Remestemcel-L Single-Arm Study For Pediatric GVHD

In advisory committee briefing documents, agency also says critical quality attributes of the graft-versus-host disease treatment do not show a relationship with clinical potency and may not ensure control of clinical effectiveness of individual drug product lots.

Stem Cells Therapy Concept stem cell transplant through perfusion — FDA panel to vote on whether data support efficacy of remestemcel-L in pediatric patients with graft-versus-host disease • Source: Shutterstock

The US Food and Drug Administration is questioning whether Mesoblast Limited’s pivotal Phase III single-arm study of Ryoncil (remestemcel-L) is sufficient to grant approval of the cellular therapy for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients.

While the study met its primary endpoint, the FDA said it does not appear to be adequate and well controlled...