FDA seeks more data about cell therapy for pediatric GVHD
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The FDA issued a complete response letter to a biologics license application that seeks approval of remestemcel-L to treat certain children with graft-versus-host disease.
The agency recommended the company developing remestemcel-L (Ryoncil, Mesoblast Limited) conduct at least one additional randomized study to provide more evidence about the therapy’s effectiveness.
Mesoblast issued a press release indicating company officials will “urgently request” a meeting with the FDA to discuss the possibility of accelerated approval for the therapy with a post-approval condition for an additional study.
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.
Remestemcel-L — an allogeneic cell therapy comprised of culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor — is administered to patients in a series of IV infusions.
Earlier this year, the FDA granted priority review to remestemcel-L for the treatment of children with steroid-refractory acute GVHD.
The agency’s Oncologic Drugs Advisory Committee (ODAC) supported approval of remestemcel-L for this indication despite concerns about limited efficacy data.
Randomized trial results 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.
“The phase 3 trial results showed that remestemcel-L provides a meaningful treatment for children with [steroid-refractory acute GVHD] who have a very dismal prognosis. I look forward to having this much-needed therapy available to our patients,” Joanne Kurtzberg, MD, director of the pediatric blood and marrow transplant program and codirector of the stem cell transplant laboratory at Duke University Medical Center, said in the Mesoblast-issued press release.
Mesoblast also is conducting a randomized phase 3 trial to evaluate remestemcel-L for ventilator-dependent adults with moderate to severe acute respiratory distress syndrome due to COVID-19. This inflammatory disease has a similar profile of damaging inflammatory cytokines as observed among children with steroid-refractory acute GVHD.
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