The EveryLife Foundation for Rare Diseases commends the Food and Drug Administration (FDA) on today’s announcement of its intention to create a Rare Disease Innovation Hub. Co-chaired by CDER Director, Dr. Peter Marks, and CBER Director, Dr. Patrizia Cavazzoni,...
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, announced today it has resubmitted its BLA for approval of Ryoncil® (remestemcel-L) in the treatment of children with SR-aGVHD. The filing comes after...
The biggest news of the week was the FDA approval of Eli Lilly’s Kisunla (donanemab) on Tuesday. While not unexpected, it was one of the year’s most highly anticipated decisions. And last week saw another big approval from the FDA—that of Verona’s novel COPD drug. But...
Company to begin enrollment of patients aged 1-3 years Expects data from younger cohort to be part of pivotal plans and BLA filing for broad label End-of-Phase II meeting with FDA scheduled for late July to finalize pivotal program design Based on recent commercial...
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), applauds the U.S. Food and Drug Administration (FDA) for its decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin...
