First-Ever Cellular Drug for Pediatric Steroid-Refractory Acute GVHD Under FDA Review

The Food and Drug Administration (FDA) accepted a resubmission for a Biologics Licensing Application for remestemcel-L to treat children with steroid-refractory acute graft-versus-host disease (SR-aGVHD), according to Mesoblast Limited, the manufacturer of the drug.

By accepting the Biologics Licensing Application, the FDA is agreeing to review the drug for potential approval, where it can be introduced into the United States Market. The agency set a goal date of Aug. 2, 2023, to make their approval decision.

aGVHD is a serious and potentially life-threatening complication of allogeneic bone marrow transplant, which is used to treat blood cancers. It is often treated with steroids, but in some instances, steroids either do not work or stop working. This group of patients with SR-aGVHD have not seen an improvement in survival outcomes in decades, highlighting the need for a new treatment, according to a press release from Mesoblast.

READ MORE: Playing ‘Whac-a-Mole’ While Managing GVHD Symptoms After Transplantation

In fact, if remestemcel-L is approved, it will be the first therapy for children aged 12 and younger with SR-aGVHD that is approved by the FDA.

The drug is an off-the-shelf cellular therapy, meaning that it is created from the cells of an unrelated donor. The manufacturers take and expand the donor’s mesenchymal stromal cells — which work to counteract the inflammatory molecules that cause GVHD — and then re-infuse them into the patient being treated.

Mesoblast Limited originally filed a Biologics Licensing Application with the data of 309 children with SR-aGVHD, showing that the cellular therapy improved treatment responses and survival. Now, the pharmaceutical company updated the data given to the FDA to include information on patient subtypes.

In a group of high-risk children treated with remestemcel-L, 67% responded positively to treatment within 28 days and were alive after 180 days. In a group of children treated with other biologic products, including Jakafi (ruxolitinib), only 10% responded and were alive after 180 days.

Also included in the resubmission was results from a four-year study on 51 patients. This data showed that in patients treated with remestemcel-L, 63% were alive at one year and 51% were alive at two years. The majority (89%) of the patients in this study had grade C/D disease, with an average two-year survival of 25-38% using best available therapy, according to the release.

“Over the last two years we have worked tirelessly to address the issues previously raised by FDA. We look forward to working closely with the Agency over the review period with the aim to make remestemcel-L available as a therapy for children suffering from SR-aGVHD,” said Mesoblast chief executive Silviu Itescu, in the press release.

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