Clinical TrialApr 10, 2026, 04:12 PM
Mesoblast Gets FDA IND Clearance for Ryoncil® DMD Trial
AI Summary
Mesoblast Limited announced that the FDA has granted Investigational New Drug (IND) clearance for a registrational clinical trial evaluating Ryoncil® in Duchenne muscular dystrophy (DMD). This trial will enroll 76 patients aged 5 to 9 years, aiming to leverage Ryoncil's anti-inflammatory mechanism to preserve muscle function and slow disease progression. The primary endpoint is time-to-stand at nine months, a validated FDA endpoint, representing a significant step forward for the company's pipeline.
Key Highlights
- FDA granted IND clearance for Ryoncil® registrational trial in Duchenne muscular dystrophy (DMD).
- DMD affects approximately 15,000 children in the U.S.
- Trial will randomize 76 patients aged 5 to 9 years to Ryoncil® or placebo.
- Primary endpoint is time-to-stand at nine months, a validated FDA endpoint.
- Mesoblast partnered with Parent Project Muscular Dystrophy for patient identification.
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