Today (10/2) the U.S. Food and Drug Administration (FDA) announced the rejection of Ryoncil, a treatment for steroid-refractory acute graft versus host disease (SR-aGVHD) in pediatric patients. For patients with the most aggressive form of SR-aGVHD, the mortality rate is 90%, and there are no current treatment options for children under 12 facing this disease. Despite these grim realities and the 9-1 vote for approval by the Oncologic Drugs Advisory Committee, the FDA requested that Mesoblast conduct one more randomized clinical trial including pediatric patients to test the functionality of the drug. Mesoblast is currently conducting a Phase 3 clinical trial on COVID-19 adult patients with similar inflammatory symptoms to push the first-of-its-kind drug to the market.
Read more here.