Longeveron (LGVN) Announces FDA Approves its Lomecel-B for Rare Pediatric Disease Designation to Treat Life-Threatening Infant Heart Condition
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 post-surgery. Other measurements of the babies’ health, such as weight gain and growth pattern, matched that of normal healthy babies.
The FDA may grant RPD designation for diseases that primarily affect children ages 18 years old or younger, and fewer than 200,000 individuals in the U.S. Under this program, if the FDA approves Lomecel-B for the treatment of HLHS, Longeveron may be eligible to receive a priority review voucher (PRV) if the application submitted for the product satisfies certain conditions, and under current law, is approved prior to September 30, 2026. Drug companies receiving a PRV can have any subsequent drug or biologic application reviewed and a decision made in six months as opposed to the standard 10 months, potentially accelerating the time to market, or they can sell the PRV to another company. The PRV accelerated review is for patients with serious conditions providing access to a potentially lifesaving or -changing treatment sooner. For companies, it means they can potentially go to market with their product more quickly, and potentially begin generating product revenue.
Infants with HLHS are currently being enrolled in ELPIS II, a 38-subject, phase 2 randomized, double-blind, controlled clinical trial. ELPIS II is being funded in part by a grant from the National Institute of Health’s National Heart, Lung, and Blood Institute (NHLBI; Grant number 1UG3HL148318), in collaboration with Longeveron, and is led by Principal Investigator Sunjay Kaushal, MD, PhD, Division Head, Cardiovascular-Thoracic Surgery, Ann and Robert H. Lurie Children’s Hospital of Chicago. Other participating clinical sites currently open for enrollment are Advocate Children’s Hospital, Park Ridge, IL; Primary Children’s Hospital/University of Utah, Salt Lake City, UT; Children’s Hospital Los Angeles, Los Angeles, CA; and Cincinnati Children’s Hospital Medical Center, Cincinnati, OH.