The US Food and Drug Administration (FDA) has approved Mesoblast Limited’s Ryoncil, a mesenchymal stromal cell therapy, as the first treatment of its kind in the country for steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older.
SR-aGvHD is a rare but serious condition that can occur after a bone marrow transplant. The FDA has only approved Ryoncil, which contains mesenchymal stromal cells isolated from healthy adult donors, for this specific indication.
In a single-arm Phase 3 trial, the therapy showed promising results with 70% of participants achieving an overall response by Day 28. This measure is associated with improved survival rates in SR-aGvHD patients.
Notably, Ryoncil treatment was completed without interruption in over 85% of patients, and no laboratory abnormalities led to discontinuation. The FDA’s approval marks a significant milestone for Mesoblast Limited.
The company’s stock price surged by 42.60% at $17.47 during the premarket session following the announcement.
Source: https://finance.yahoo.com/news/mesoblasts-cell-therapy-treatment-graft-113808474.html