Investing.com -- Shares of Mesoblast (NASDAQ:MESO) Limited surged by 39% in pre-open trading Thursday following the Food and Drug Administration's (FDA) approval of Ryoncil® (remestemcel-L), marking the first mesenchymal stromal cell (MSC) therapy to receive the green light in the United States. The regulatory nod positions Ryoncil as the sole 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 serious and potentially fatal condition.
The FDA's decision was influenced by the outcomes of a Phase 3 trial, where 70% of children with high-severity SR-aGvHD achieved an overall response by Day 28 of treatment with Ryoncil. This response rate is particularly significant as it serves as a predictor of survival in patients with aGvHD. New York-based Mesoblast, a global leader in allogeneic cellular medicines for inflammatory diseases, has now successfully introduced the first MSC product to the U.S. market.
Dr. Joanne Kurtzberg, a renowned transplant physician, commented on the gravity of this breakthrough, stating, “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.”
This approval is a significant milestone for Mesoblast, as it opens doors to potentially other indications in diseases characterized by excessive inflammation, given RYONCIL’S immunomodulatory effects. The company's CEO, Dr. Silviu Itescu, expressed satisfaction with the FDA's approval and reaffirmed Mesoblast's commitment to address the unmet needs of the GVHD community and to continue pursuing approval for other late-stage products.
RYONCIL is slated to be available in the United States at transplant centers and other treating hospitals, providing a new treatment option for the approximately 1,500 children in the U.S. who develop SR-aGvHD annually after an allogeneic bone marrow transplant. The therapy's recommended dosage involves intravenous infusions given twice per week for four consecutive weeks, with the response assessed 28 ± 2 days after the initial dose.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.