Mesoblast submits therapy to the US FDA

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Mesoblast (ASX:MSB), a company focused on the development of cellular medicines for inflammatory diseases, has submitted its completed Biologics License Application (BLA) to the US Food and Drug Administration for RYONCIL (remestemcel-L).

RYONCIL is an investigational therapy comprising culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is for the treatment of children with steroid-refractory acute graft versus host disease (SRaGVHD) and administered via a series of intravenous infusions.

The company said it has requested Priority Review of the BLA by the FDA under the product candidate’s existing Fast Track designation for SR-aGVHD. If approved, RYONCIL is expected to be launched in the US in 2020.

According to Mesoblast chief executive Dr Silviu Itescu, “This is a major corporate milestone for Mesoblast. We look forward to working closely with the FDA to potentially bring RYONCIL to market and providing our innovative biologic therapy to the many children with this life-threatening condition.”

Acute GVHD occurs in approximately 50 per cent of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers.

Mortality is high, up to 90 per cent, in patients with the most severe form of acute GVHD. There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.