FDA Approves Compassionate Use of Longeveron Therapy in Child with Rare Heart Defect
March 11, 2021
Rare Daily Staff
The U.S. Food and Drug Administration has granted expanded access approval for the administration of Longeveron’s experimental cell therapy Lomecel-B to a child with hypoplastic left heart syndrome, a rare heart defect.
Hypoplastic left heart syndrome (HLHS) is a rare congenital heart defect that effects approximately 1,000 babies per year in the United States. 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-stage 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.
Lomecel-B is an allogeneic, bone marrow-derived medicinal signaling cell (MSC). Sunjay Kaushal, division head of Cardiovascular Thoracic Surgery at the 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 experimental 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 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,” said Kaushal, who was the first surgeon in the United States to administer allogeneic MSCs to a baby with HLHS. “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.”
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. 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. Kaushal is the principal investigator of the study, which is funded by the National Heart, Lung and Blood Institute.
“Our goal is to provide a new way to treat HLHS,” said Geoff Green, CEO of Longeveron. “We believe, based on previous studies, that the MSCs in Lomecel-B may improve ventricular and vascular function.”

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