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U.S. Food and Drug Administration Approves Longeveron’s Lomecel-B for Rare Pediatric Disease Designation to Treat Life-Threatening Infant Heart Condition
Phase 2 clinical trial underway for Hypoplastic Left Heart Syndrome, which affects approximately 1,000 babies per year MIAMI, Nov. 18, 2021 (GLOBE NEWSWIRE)
About this update from Longeveron Inc.
[{"type":"text","content":"Phase 2 clinical trial underway for Hypoplastic Left Heart Syndrome, which affects approximately 1,000 babies per year\nMIAMI, Nov. 18, 2021 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) (\"Longeveron\" or \"Company\"), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and life-threatening conditions, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants. Lomecel-B, an investigational allogeneic, bone marrow-derived medicinal signaling cell (MSC) product, is currently 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.” Approximately 1,000 babies are born with HLHS each year in the U.S. HLHS babies have an underdeveloped left ventricle, which impairs the heart’s ability to pump blood throughout the body. HLHS is often fatal without surgical intervention, in which three surgical procedures are performed over the period of about 5 years, to allow the right ventricle to be configured to pump blood to the body. Longeveron is evaluating the safety of Lomecel-B injection into the right ventricle during the second surgery (4 – 6 months of age), and the effect on cardiac function and other health status endpoints. Longeveron recently reported clinical results from its safety-focused Phase I clinical study of Lomecel-B in HLHS patients. When cardiac surgeons injected Lomecel-B directly into the babies’ hearts at the time of surgery, the cells were well tolerated with no major adverse cardiac events and no infections considered to be related to the investigational treatment. One hundred percent of infants enrolled in the Phase 1 trial (n=10) were alive and had not required a transplant between 2 – 3.5 years ...