The US Food and Drug Administration (FDA) has approved Ryoncil, a treatment for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients over 2 months old. This rare blood disease can cause significant health consequences, including organ damage and risk of death.
Ryoncil is the first FDA-approved cell therapy for this condition, using mesenchymal stromal cells from healthy adult donors. The treatment involves intravenous infusion twice a week for four weeks, with monitoring by treating physicians.
According to the FDA, 16 out of 54 study participants had a complete response to treatment, and 22 had a partial response. Common side effects include infections, fever, and hypotension.
The approval is an important step in addressing unmet needs for patients with debilitating diseases. The FDA granted Orphan Drug, Fast Track, and Priority Review designations to Mesoblast, Inc., the developer of Ryoncil.
Source: https://www.prnewswire.com/news-releases