Mesoblast announced that the United States Food and Drug Administration has granted Investigational New Drug (IND) clearance to directly proceed to a registrational clinical trial evaluating Ryoncil (remestemcel-L-rknd) in Duchenne muscular dystrophy.
The randomized trial will enroll 76 patients aged 5 to 9 years, assigning participants to Ryoncil (7 infusions of 2 × 10^6 cells per kg over 9 months) or placebo in addition to standard of care, with the primary endpoint of time-to-stand at 9 months, a validated FDA endpoint for approval.
According to the company, Ryoncil is the first mesenchymal stromal cell product approved by the FDA and the only product approved for children under 12 with steroid-refractory acute graft-versus-host disease. The company claims Ryoncil’s anti-inflammatory mechanism may reduce the inflammatory cascade in DMD, preserve muscle function and slow disease progression.
Mesoblast will work with Parent Project Muscular Dystrophy and the Duchenne Registry to support patient identification and trial awareness. The press release corrects an April 7 version and clarifies that it was not intended to imply endorsement by PPMD. The company notes DMD affects approximately 15,000 children in the United States.
