FDA panel supports Ryoncil cell therapy for children with acute GVHD
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An FDA advisory committee voted 9-1 in favor of approval of the cell therapy remestemcel-L for the treatment of children with steroid-refractory acute graft-versus-host disease.
The FDA’s Oncologic Drugs Advisory Committee (ODAC) supported approval despite concerns about limited efficacy data for remestemcel-L (Ryoncil, Mesoblast Ltd.).
Results of randomized trials showed the therapy failed to confer significant benefit; however, those results were among adults rather than children.
Results of a single pediatric arm of one phase 3 clinical trial showed an acceptable overall response rate and duration of response, according to ODAC panelists. In that cohort, 69% of pediatric patients who received remestemcel-L achieved complete or partial response after 28 days.
ODAC panelists also expressed concerns about manufacturing, including use of a complicated process that can lead to inconsistent efficacy between cell batches.
Remestemcel-L — which comprises culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor — is administered to patients in a series of IV infusions.
Approximately half of patients who undergo allogeneic bone marrow transplantation develop acute GVHD. There are no FDA-approved treatments in the United States for children aged younger than 12 years who develop steroid-refractory acute GVHD.
ODAC’s recommendation does not guarantee approval for remestemcel-L. The FDA often follows the guidance of the advisory committee when deciding on drug applications, but the agency is not required to do so.
As previously reported by Healio, the FDA granted priority review to remestemcel-L. A decision on approval is expected by Sept. 30.
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