Mesoblast Initiates Rolling Submission of Biologics License Application to FDA

 Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in cellular medicines for inflammatory diseases, today announced that it has filed the first component of a rolling submission for a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease (aGVHD), a life-threatening complication of an allogeneic bone marrow transplant.

The FDA has agreed to a rolling review of the BLA which enables individual components to be submitted and reviewed on an ongoing basis rather than waiting for all sections to be completed. The rolling process will provide opportunity for ongoing communication, and during this process the Company expects it will be able to adequately address any substantial matters raised by the FDA. Remestemcel-L has received Fast Track designation for aGVHD and under this designation Mesoblast intends to request a priority review once its BLA filing is completed and accepted by the FDA.

Mesoblast Chief Executive Dr Silviu Itescu stated: “Initiation of our rolling BLA submission to the FDA for potential United States approval of our first product is a major corporate milestone for the Company and an exciting moment in our history. We look forward to making this therapy available as soon as possible to children with this devastating disease.”

There are more than 30,000 allogeneic bone marrow transplants performed annually worldwide¹, primarily in patients being treated for blood cancers, with up to 50% developing aGVHD. In the more severe forms of the disease, Grades C/D or III/IV, six-month mortality rates are as high as 90%.^2,3 

In Mesoblast’s Phase 3 trial of 55 children with aGVHD – 89% of whom had Grade C/D disease – treatment with remestemcel-L resulted in a six-month survival of 69%. In addition, achievement of an Overall Response at Day 28, which occurred in 69% of patients, predicted highest survival at Day 100 and Day 180, which was 85% and 79%, respectively. The trial successfully met its primary endpoint of increased Day 28 Overall Response compared with a protocol-defined historical control rate of 45% (p=0.0003). These data are consistent with prior results from an Expanded Access Program in 241 children where remestemcel-L was used as salvage therapy after failure of steroids and other agents.

https://www.globenewswire.com/news-release/2019/05/30/1856806/0/en/Mesoblast-Initiates-Rolling-Submission-of-Biologics-License-Application-BLA-to-U-S-FDA-for-remestemcel-L-in-the-Treatment-of-Acute-Graft-Versus-Host-Disease.html