FDA clears Mesoblast Duchenne trial for disease affecting 15,000 U.S. children

Mesoblast has received FDA Investigational New Drug (IND) clearance to directly proceed with a registrational trial for Ryoncil in Duchenne muscular dystrophy (DMD). The trial will enroll 76 patients aged 5-9, with time-to-stand at nine months as the primary endpoint. This builds on Ryoncil’s existing pediatric safety profile, preclinical efficacy in DMD models, and an FDA-approved manufacturing process.