Longeveron Inc. (NASDAQ: LGVN) has received approval for Rare Pediatric Disease Designation for Lomecel-B by the FDA to treat Hypoplastic Left Heart Syndrom. HLHS is a rare life-threatening congenital heart defect in infants. Lomecel-B is an investigational allogeneic bone marrow derived medicinal signaling cell product that is being evaluated in a Phase 2 trial. “We are pleased by FDA’s acknowledgment of the urgent need for a safe and effective treatment for children born with this rare and devastating congenital heart defect,” stated Longeveron co-founder and Chief Science Officer Joshua M. Hare, M.D. “We are encouraged by our Phase 1 clinical data, and the progress being made in the ongoing Phase 2 trial. Lomecel-B represents a unique cell therapy approach that could potentially be administered at the same time as surgery in these critically impacted infants.”
About 1,000 babies are born with HLHS each year within the United States. HLHS babies have an underdeveloped left ventricle that impairs the heart’s ability to pump blood throughout the body. HLHS is fatal without surgical intervention which requires three surgical procedures performed over a period of 5 years to allow theright ventricle to be configured to pump blood to the body. Longeveron is currently evaluating the safety of Lomecel-B in HLHS patients. Cardiac surgeons inject Lomecel-B directly into the babies’ hearts at time of surgery, the cells were tolerated with no major adverse cardiac events and no infections that are considered to be related to the investigational treartment.