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

 
• By Brenda Sandburg

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 given the absence of appropriate concurrent or historical controls and thus may not be sufficient to

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