FDA greenlights Longeveron IND for MSC therapy in pediatric heart condition

The U.S. FDA has approved Longeveron’s IND application for its mesenchymal stem cell therapy, laromestrocel, as a potential treatment for pediatric dilated cardiomyopathy (DCM), a rare, life-threatening disease with no current effective treatment.

The accepted IND application will allow Longeveron to move directly to a single phase 2 pivotal registration clinical trial, which the company plans to kick off in first half of 2026.

“With nearly 40% of children with DCM requiring a heart transplant or dying within two years of diagnosis, there is a tremendous need for novel approaches to treat this pediatric cardiovascular disease,” said Nataliya Agafonova, MD, Longeveron’s chief medical officer.

Laromestrocel is an allogeneic mesenchymal stem cell therapy product isolated from the bone marrow of young, healthy adult donors. Laromestrocel has multiple potential mechanisms of action that may lead to anti-inflammatory, pro-vascular and regenerative responses, and therefore broad potential applications across a spectrum of disease areas.