The Food and Drug Administration (FDA) has granted Orphan Drug designation to Lomecel-B for the treatment of hypoplastic left heart syndrome, a rare, life-threatening congenital heart defect in which the left ventricle is either severely underdeveloped or missing.

Lomecel-B is derived from culture-expanded medicinal signaling cells that are sourced from the bone marrow of young, healthy adult donors. It is delivered via intramyocardial injection.

The designation is supported by data from a safety-focused phase 1 clinical study ( Identifier: NCT03525418) evaluating Lomecel-B in 10 HLHS infants. Patients were treated with Lomecel-B at the time of the second surgery.

Findings showed that all of the infants were alive and did not require a transplant 2 to 3.5 years after surgery. Lomecel-B was found to be well tolerated; no major adverse cardiac events or infections related to the investigational therapy were reported.

The Company is currently investigating the effects of Lomecel-B in conjunction with reconstructive surgery in infants with HLHS in the phase 2 ELPIS II trial ( Identifier: NCT04925024).

“Building on results from our completed phase 1 safety-focused trial, we believe Lomecel-B has potential to improve outcomes for these severely impacted infants by way of repairing cardiac tissue and improving ventricular function,” said Geoff Green, CEO at Longeveron.

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.


Longeveron granted Orphan Drug designation by FDA for Lomecel-B to treat infants with hypoplastic left heart syndrome (HLHS). News release. Longeveron Inc. Accessed December 6, 2021.