Mesoblast resubmits remestemcel-L BLA to treat steroid-refractory acute graft versus host disease

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According to Mesoblast Limited, the BLA resubmission is expected to have a review period of between 2 and 6 months from receipt.

Mesoblast resubmits remestemcel-L BLA to treat steroid-refractory acute graft versus host disease | Image Credit: © Postmodern Studio - © Postmodern Studio - stock.adobe.com.

Mesoblast resubmits remestemcel-L BLA to treat steroid-refractory acute graft versus host disease | Image Credit: © Postmodern Studio - © Postmodern Studio - stock.adobe.com.

The Biologics License Application (BLA) for remestemcel-L (Ryoncil; Mesoblast Limited) has been resubmitted to the FDA as a potential treatment for children with steroid-refractory acute graft versus host disease (SR-aGVHD), according to a press release from Mesoblast Limited.1

The filing follows the FDA informing Mesoblast that available clinical data from the phase 3 study, MSB-GVHD001, "appears sufficient to support submission of the proposed BLA for remestemcel-L for treatment of pediatric patients with SR-aGVHD," the company stated in the press release. The filing addresses the remaining chemistry, manufacturing, and control items.1

"We have worked closely with the agency and thank them for their ongoing guidance, facilitating the potential approval of RYONCIL and addressing the urgent need for a therapy that improves the dismal survival outcome in children with SR-aGVHD,” Silviu Itescu, CEO, Mesoblast Limited, said in a statement.1

In June 2024, the company announced a resubmission was in the works, following the issuance of a Complete Response Letter (CRL) from the FDA on August 4, 2023, at the time noting more data was needed to support the marketing approval.2,3

At the time of CRL issuance, the FDA completed the Pre-License Inspection and did not issue any Form 483. No objectionable conditions were found, and the federal agency “acknowledged in the resubmission review that changes implemented appear to improve assay performance relative to the original version of the assay used in the pediatric phase 3 trial,” according to previous coverage on Contemporary Pediatrics.2

In a single-arm, phase 3 trial that enrolled 54 children with SR-aGVHD, a pre-specified primary outcome was met. In August 2020, the Oncologic Drugs Advisory Committee voted 9 to 1 in favor of remestemcel-L’s pediatric population efficacy. In September of the same year, the FDA noted that additional steps were needed for approval. The January 2023 BLA resubmission for remestemcel-L included long-term follow-up data from the Center for International Blood and Marrow Transplant Research phase 3 trial.2

Fifty-percent survival through 4 or more years during follow up in remestemcel-L-treated patients was observed in the phase 3 trial. According to Mesoblast, less than 20% survival was expected based on severity of disease. A post-hoc propensity matched study was also included in the resubmission, demonstrating that 6-month survival was 67% with remestemcel-L compared to 10% with other non-approved therapies.2

These results were demonstrated in highest-risk patients (identified using Mount Sinai Acute GVHD International Consortium). These data further support remestemcel-L use for the proposed study to treat high-risk adults with SR-aGVHD, stated Mesoblast.2

Remestemcel-L was granted Fast Track designation by the FDA, a process that facilitates development and expedited review of therapies for serious conditions that full unmet medical needs. The potential treatment was also granted Priority Review designation.1

According to Mesoblast Limited, the BLA resubmission is expected to have a review period of between 2 and 6 months from receipt.1

References:

  1. Mesoblast Resubmits Biologics License Application (BLA) with United States Food & Drug Administration (FDA) for Approval of Ryoncil® in Children with Steroid-Refractory Acute Graft-Versus-Host Disease (SR-aGVHD). Mesoblast Limited. Press release. July 9, 2024. Accessed July 9, 2024. https://www.biospace.com/article/releases/mesoblast-resubmits-biologics-license-application-bla-with-united-states-food-and-drug-administration-fda-for-approval-of-ryoncil-in-children-with-steroid-refractory-acute-graft-versus-host-disease-sr-agvhd-/
  2. Fitch, J. BLA for steroid-refractory acute graft versus host disease candidate to be resubmitted. Contemporary Pediatrics. June 4, 2024. Accessed July 9, 2024. https://www.contemporarypediatrics.com/view/bla-for-steroid-refractory-acute-graft-versus-host-disease-candidate-to-be-resubmitted
  3. Fitch, J. FDA issues complete response letter to remestemcel-L for graft-versus-host disease treatment. Contemporary Pediatrics. August 10, 2023. Accessed July 9, 2024. https://www.contemporarypediatrics.com/view/fda-issues-complete-response-letter-to-remestemcel-l-for-graft-versus-host-disease-treatment
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