FDA grants IND clearance to Mesoblast for a registrational trial of Ryoncil (remestemcel-L-rknd) in children with Duchenne muscular dystrophy, evaluating anti-inflammatory MSC therapy across 76 patients.
Written By: A. Musharaf Mohammad, BPharm
Reviewed By: Pharmacally Editorial Team
Mesoblast Limited announced that the U.S. Food and Drug Administration has granted Investigational New Drug clearance to proceed directly to a registrational clinical trial of Ryoncil (remestemcel-L-rknd) for children with Duchenne Muscular Dystrophy. The condition affects approximately 15,000 children in the United States.
Ryoncil is a mesenchymal stromal cell therapy and is currently the first MSC product approved by the FDA, indicated for children under 12 years with steroid-refractory acute graft-versus-host disease. Mesoblast plans to leverage the product’s anti-inflammatory mechanism to address the inflammatory cascade that contributes to muscle degeneration in Duchenne muscular dystrophy, with the goal of preserving muscle function and slowing disease progression.
The registrational study will randomize 76 patients aged 5 to 9 years to receive either Ryoncil or placebo in addition to standard of care. Patients assigned to the treatment arm will receive seven infusions of 2 × 10⁶ cells/kg over nine months. The primary endpoint will be time-to-stand at nine months, a functional outcome accepted by the FDA as a validated endpoint for approval.
To support recruitment and patient identification, Mesoblast is collaborating with Parent Project Muscular Dystrophy, a leading patient advocacy organization founded in 1994, to enhance patient identification, education, and trial awareness through proactive community engagement.
The study’s principal investigator, Aravindhan Veerapandiyan, MD, stated that targeting inflammation in Duchenne muscular dystrophy may help intervene while muscle tissue is still preserved, potentially modifying disease progression. He noted that Ryoncil’s anti-inflammatory activity could address a major driver of deterioration in affected children.
Mesoblast Chief Executive Silviu Itescu said the FDA clearance to proceed directly to a registrational study was supported by preclinical data in Duchenne muscular dystrophy models and extensive pediatric safety experience in steroid-refractory acute graft-versus-host disease. He added that the company believes the therapy may offer a new approach for children with this progressive disease.
Duchenne muscular dystrophy is an X-linked genetic disorder caused by the absence of functional dystrophin, leading to progressive degeneration of skeletal, respiratory, and cardiac muscles. The disease primarily affects boys and results in loss of ambulation, respiratory failure, and cardiomyopathy, with most patients dying by the third decade of life. While gene therapies targeting dystrophin are advancing, persistent inflammation remains a key contributor to disease progression, and long-term corticosteroid therapy is limited by plateauing benefit and safety concerns.
Reference
Mesoblast receives IND clearance from FDA to directly proceed to registrational trial for approval of RYONCIL® in Duchenne Muscular Dystrophy, 08 April 2026, https://investorsmedia.mesoblast.com/static-files/385a9c65-9fa5-4160-9c54-7dcd8e4d88ca
About the Writer
Mohamed Musharaf A is a Pharmacy graduate from Ariyur, Puducherry, with a strong interest in data analysis and its application in healthcare. He is particularly interested in using data-driven insights to support pharmacovigilance and medical writing, with a focus on understanding complex datasets and translating them into meaningful, actionable solutions. Known for his dedication to learning and attention to detail, he continuously works to strengthen his analytical and domain knowledge.