Mesoblast Resubmits BLA For Ryoncil Approval

The globally recognized allogeneic cellular therapy developer, Mesoblast Limited, has resubmitted its BLA for the approval of Ryoncil to treat children with SR-aGVHD.

Following further evaluation, the FDA notified Mesoblast at the end of March that the Phase 3 study MSB-GVHD001’s accessible clinical information seemed adequate to warrant the delivery of the proposed BLA for remestemcel-L to treat individuals with SR-aGVHD. Consequently, the submission addresses the remaining CMC (Chemistry, Manufacturing, and Control) issues.

Dr. Silviu Itescu, CEO of Mesoblast, stated, “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.”

The FDA has given Fast Track designation to remestemcel-L, a mechanism intended to speed up the development and prompt assessment of therapies targeting severe conditions that address significant medical needs. Moreover, remestemcel-L has been granted Priority Review designation, which is reserved for drugs that manage serious illnesses and demonstrate a considerable enhancement in safety or effectiveness over existing treatments.

Upon the acceptance of the Biologics License Application (BLA) resubmission, the FDA is expected to review the application within a timeframe of two to six months from the date of receipt. This expedited review process underscores the urgency and potential impact of remestemcel-L in addressing critical health challenges.

The Phase 3 GVHD001/002 study involved 54 children—89% of whom were classified as Grade C/D—across 20 centers in the U.S., where Ryoncil was administered as the initial treatment for those who did not respond to steroids for acute graft-versus-host disease (GVHD). The trial achieved its predetermined primary goal, demonstrating a Day 28 Overall Response (OR) rate of just over 70% compared to 45%, with a p-value of 0.0003.

Achieving an overall response at Day 28 was a strong predictor of enhanced survival rates through Day 100, with 87% survival in responders versus 47% in non-responders.

Acute GVHD affects about half of individuals who receive an allogeneic bone marrow transplant (BMT). Each year, over 30,000 patients worldwide undergo an allogeneic BMT, mainly as a treatment for blood cancers, with approximately 20% of these patients being children. SR-aGVHD has a mortality rate as high as 90% and leads to high costs associated with hospital stays.

Currently, there are no FDA-approved treatments available in the country for children under the age of 12 with SR-aGVHD.

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