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.
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Mesoblast’s Cell Therapy For Pediatric GVHD Gets US Panel Green Light Despite FDA's "Substantial Concerns"
Oncologic Drugs Advisory Committee votes 8-2 that data support efficacy of the product, which could be the first mesenchymal stem cell product to clear the agency. FDA's concerns include reliance on a pivotal Phase III single-arm study.
Lawyer asks US FDA to require another trial before licensing Mesoblast’s stem cell therapy for children with SR-aGVHD; Incyte’s Jakafi is only product approved for the indication.
FDA concluded that original formulation of brain hemorrhage treatment was not discontinued for safety or effectiveness reasons, saying Arbor had other options to reduce risk of confusion between the new and original formulations. But the sponsor argues that withdrawal was part of FDA-approved risk mitigation plan.