Mesoblast RYONCIL® Therapy Approved by FDA

• RYONCIL® (remestemcel-L) becomes the first mesenchymal stromal cell (MSC) product approved by the US Food and Drug Administration (FDA) for any indication.
• RYONCIL® is also the first FDA-approved therapy for children aged 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR-aGvHD), a life-threatening condition with high mortality rates.

Mesoblast (ASX:MSB; Nasdaq:MESO) has rallied strongly in what has been a down day for the overall market, after announcing that the US Food and Drug Administration (FDA) had approved the company’s RYONCIL® (remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the US.

It means that RYONCIL® is the only MSC therapy approved in the US for any indication, and the only approved therapy for steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older, including adolescents and teenagers. The latter disease is a life-threatening condition with high mortality rates.

In a single-arm, multi-center, Phase 3 trial of children with SR-aGvHD, 89% of whom had high severity Grade C or Grade D disease, 70% achieved an overall response by Day 28 of treatment with RYONCIL®, a measure that predicts survival in aGVHD.

RYONCIL®’s immunomodulatory effects, including inhibition of T cell activation and secretion of pro-inflammatory cytokines, position the therapy for potential other indications in diseases with excessive inflammation.

RYONCIL® will be available in the US at transplant centers and other treating hospitals.

Transplant physician Dr Joanne Kurtzberg, the Jerome Harris Distinguished Professor of Pediatrics and Professor of Pathology, and Director, Marcus Center for Cellular Cures at Duke University Medical Center (DUMC), said: “Steroid-refractory acute graft versus host disease is a devastating condition with an extremely poor prognosis. From today we are able to offer RYONCIL®, the first FDA-approved treatment which will be life saving for so many children and will have a great impact on their families.”

Dr. Silviu Itescu, Chief Executive of Mesoblast said: ““We are very pleased that the FDA has granted approval of RYONCIL® and are proud of the company’s commitment to the GVHD community in bringing this important new treatment to children and families with no other acceptable options.

With RYONCIL® approval by FDA, Mesoblast has demonstrated the ability to bring the first MSC product to market. We will continue to work closely with FDA to obtain approval of our other late-stage products, including REVASCOR® for cardiovascular diseases and rexlemestrocel-L for inflammatory pain indications, as well as expanding the indications for RYONCIL® in both children and adults with inflammatory conditions.”

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Tim Grey

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