Longeveron Inc. (NASDAQ:LGVN) saw its stock climb 23% after receiving clearance from the U.S. Food and Drug Administration (FDA) to begin a Phase 2 clinical trial of its investigational therapy, laromestrocel, for treating pediatric dilated cardiomyopathy (DCM).
The FDA approved Longeveron’s Investigational New Drug (IND) application, allowing the biotech company to move straight into a pivotal Phase 2 registration trial, skipping earlier stages of clinical testing. The Miami-based firm plans to launch the study in the first half of 2026.
Laromestrocel is a cellular therapy based on mesenchymal stem cells and is being developed to address pediatric DCM—a rare but serious condition that weakens the heart’s ability to pump blood due to enlarged or stretched heart muscle chambers. The disorder affects an estimated 100,000 children globally.
“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, Chief Medical Officer of Longeveron.
There are currently few treatment options for pediatric DCM, which is the most common type of cardiomyopathy in children. It accounts for roughly 50–60% of all pediatric cardiomyopathy diagnoses and is most often found in infants under one year old, with a higher prevalence in boys.
In addition to pediatric DCM, Longeveron is also advancing clinical programs targeting hypoplastic left heart syndrome, Alzheimer’s disease, and aging-related frailty. The laromestrocel platform has earned five separate FDA designations for its potential use in various conditions.