Mesoblast Shares Surge Following FDA Approval of Ryoncil

Investing.com -- Mesoblast Limited's shares surged 39% in pre-market trading on Thursday following the Food and Drug Administration's (FDA) approval of Ryoncil® (remestemcel-L). This approval marks the first green light for a mesenchymal stromal cell (MSC) therapy in the United States. The regulatory approval positions Ryoncil as the FDA-approved sole treatment for steroid-resistant acute graft versus host disease (SR-aGvHD) in children aged 2 months and older, as well as in adolescents and young adults. SR-aGvHD is a serious and potentially life-threatening condition.

The FDA's decision was influenced by the results of a Phase 3 trial. In this trial, 70% of children with severe SR-aGvHD achieved a general response by day 28 of treatment with Ryoncil. This response rate is particularly significant as it serves as an indicator of survival in patients with aGvHD. New York-based Mesoblast, a global leader in allogeneic cellular therapies for inflammatory diseases, has now successfully introduced its first MSC product to the U.S. market.

Renowned transplant doctor Dr. Joanne Kurtzberg emphasized the importance of this breakthrough, stating, "Steroid-resistant acute graft versus host disease is an incredibly devastating condition with a very poor prognosis. From today, we can offer RYONCIL, the first FDA-approved treatment that will be life-saving for many children and have a tremendous impact on their families."

This approval represents a significant milestone for Mesoblast, as RYONCIL's immunomodulatory effects potentially open doors for indications in other diseases characterized by excessive inflammation. The company’s CEO, Dr. Silviu Itescu, expressed his satisfaction with the FDA's approval and reiterated Mesoblast's commitment to addressing the unmet needs of the GVHD community while continuing to seek approval for other late-stage products.

Ryoncil is expected to be made available at transplant centers and other treatment hospitals across the United States. This offers a new treatment option for approximately 1,500 U.S. children who develop SR-aGvHD annually following allogeneic bone marrow transplant. The recommended dosage of the therapy involves intravenous infusions administered twice a week for four consecutive weeks, and responses will be evaluated 28 ± 2 days after the first dose.