Lomecel-B Approved for Compassionate Use for Child with Hypoplastic Left Heart Syndrome

Longeveron announced the U.S. Food & Drug Administration (FDA) has granted expanded access approval for the administration of Longeveron’s investigational cell therapy Lomecel-B to a child with Hypoplastic Left Heart Syndrome (HLHS). Lomecel-B is an allogeneic, bone marrow-derived medicinal signaling cell (MSC) product manufactured under cGMP in Longeveron’s cell processing facility in Miami, Florida. Dr. Sunjay Kaushal, MD, PhD, Division Head Cardiovascular Thoracic Surgery at Ann & Robert H. Lurie Children’s Hospital of Chicago will administer Lomecel-B during a reconstructive cardiac surgery procedure.

FDA’s Expanded Access program, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside of clinical trials. The Lurie Children’s Hospital Internal Review Board (IRB) also reviewed and approved the protocol.

"The rationale for this approach is to improve the functioning of the right ventricle, the only ventricle in these babies, through regeneration of cardiac tissue. Our goal is to make it pump as strongly as a normal left ventricle," Dr. Kaushal said. "We are grateful to FDA, Lurie Children’s Hospital IRB, and Longeveron for making this happen, and we are hoping this therapy will be a game-changer for this baby and others in the future.”

“Our goal is to provide a new way to treat HLHS and we believe, based on previous studies, that the MSCs in Lomecel-B may improve ventricular and vascular function,” said Geoff Green, CEO of Longeveron.

HLHS is a rare congenital heart defect that effects approximately 1,000 babies per year in the U.S. Babies with HLHS are born with an underdeveloped left ventricle, which impairs the heart’s ability to pump adequate amounts of blood throughout the body. Without a three-staged reconstructive surgery, the condition is often fatal. Even with surgery, HLHS is still associated with high mortality and a high rate of cardiac failure necessitating heart transplantation.

Recently, Longeveron announced the successful completion of its Phase 1 clinical study of Lomecel-B intramyocardial injection in HLHS patients. The study was conducted by a consortium of pediatric cardiac surgeons at centers that included the University of Maryland Medical Center, University of Cincinnati/Children’s Hospital Medical Center and University of Utah Primary Children’s Hospital, and was supported in part by a Maryland Stem Cell Research Fund TEDCO Grant. The intramyocardial injection of Lomecel-B was well-tolerated, with no major cardiac events, and no serious adverse events related to Lomecel-B were reported. The Phase 1 safety results have enabled Longeveron to advance its HLHS program into a Phase 2 multi-center trial, with a randomized, double-blind, placebo-controlled trial scheduled to begin in the third quarter of 2021, and is led by Dr. Kaushal as the Principal Investigator, and funded by the National Heart, Lung and Blood Institute.