Longeveron’s Lomecel-B™ Approved by FDA for Compassionate Use for the Treatment of Child with Hypoplastic Left Heart Syndrome (HLHS)
Longeveron Inc. (NASDAQ: LGVN) received FDA approval for expanded access to its investigational cell therapy, Lomecel-B, for a child with Hypoplastic Left Heart Syndrome (HLHS). The therapy aims to enhance the right ventricle's function, which is crucial for HLHS patients. Dr. Sunjay Kaushal of Ann & Robert H. Lurie Children’s Hospital will administer the treatment during surgery. Lomecel-B is derived from bone marrow and produced in Miami. The company successfully completed a Phase 1 study demonstrating safety, leading to a Phase 2 trial set for Q3 2021, progressing towards regulatory approval.
- FDA granted expanded access for Lomecel-B therapy for HLHS, indicating potential for broader application.
- The successfully completed Phase 1 study showed Lomecel-B was well-tolerated with no major cardiac events reported.
- Transition to a Phase 2 multi-center trial scheduled for Q3 2021 indicates progress in clinical development.
- None.
MIAMI, March 11, 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 & 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.
Dr. Kaushal was the first surgeon in the United States to administer allogeneic MSCs to a baby with HLHS. "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," says Dr. Kaushal. "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,” stated 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 leading 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.
About Longeveron Inc.
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. Longeveron believes that by using the same cells that promote tissue repair, organ maintenance, and immune system function, it can develop safe and effective therapies for some of the most difficult disorders associated with the aging process and other medical disorders. Longeveron is currently sponsoring Phase 1 and 2 clinical trials in the following indications: Aging Frailty, Alzheimer’s disease, the Metabolic Syndrome, Acute Respiratory Distress Syndrome (ARDS), and hypoplastic left heart syndrome (HLHS). The Company’s mission is to advance Lomecel-B and other cell-based product candidates into pivotal Phase 3 trials, with the goal of achieving regulatory approvals, subsequent commercialization and broad use by the healthcare community. Additional information about the Company is available at www.longeveron.com.
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