Longeveron gets FDA approval for pediatric heart disease trial

Longeveron Inc. (NASDAQ: LGVN) announced that the U.S. Food and Drug Administration approved its Investigational New Drug application for laromestrocel as a potential treatment for pediatric dilated cardiomyopathy. The approval allows the company to proceed directly to a Phase 2 pivotal registration clinical trial.

The Miami-based biotechnology company expects to initiate the Phase 2 clinical trial in the first half of 2026, according to the company statement. Laromestrocel is an investigational cellular therapy derived from mesenchymal stem cells.

Pediatric dilated cardiomyopathy affects at least 100,000 children worldwide and represents the most common form of cardiomyopathy in children. The condition occurs when heart chamber muscles become enlarged or stretched, causing the heart to weaken and struggle to pump blood effectively.

Current treatment options are limited, with nearly 40% of children with the condition requiring a heart transplant or dying within two years of diagnosis. About 50 to 60 percent of all pediatric cardiomyopathy cases are diagnosed as dilated, with the condition being more common in boys than girls and more frequent in infants under age one.

"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.

Longeveron is currently pursuing three pipeline indications: hypoplastic left heart syndrome, Alzheimer's disease, and aging-related frailty. The company's laromestrocel programs have received five FDA designations across different indications.

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