Longeveron Shares Up After FDA Approval of Lomecel-B for Rare Pediatric Disease Designation

Nov 18, 2021 By MarketDepth

Biotechnology Business Headlines Healthcare Investing What's Hot

FDA Approved

Longeveron Inc. (NASDAQ: LGVN) shares skyrocketed more than 140% after the clinical stage biotechnology company announced 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.

1,000 Babies Born Annually with Rare Disease

According to data provided by Longeveron, 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.

“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. 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.”

Longeveron co-founder and Chief Science Officer Joshua M. Hare, M.D.

The Company also recently reported clinical results from its safety-focused Phase I clinical study of Lomecel-B in HLHS patients. Longeveron indicated that 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.

Developing Cellular Therapies

Longeveron is a clinical stage biotechnology company developing cellular therapies for specific aging-related and life-threatening conditions. The Company’s lead investigational product is the LOMECEL-B™ cell-based therapy product (“Lomecel-B”), which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young, healthy adult donors.