FDA grants rare pediatric disease designation to novel allogeneic cell therapy for HLHS

Written by Alice Soteriou


HLHS is a fatal rare congenital heart defect in infants. The FDA has recently granted rare pediatric disease (RPD) designation to Lomecel-B, from  Longeveron (FL, USA). Lomecel-B is an allogeneic cell therapy, derived from bone marrow, that is currently in phase II clinical trials. 

HLHS occurs as a result of the underdevelopment or lack of the heart’s left ventricle, thereby impairing the heart’s ability to circulate blood around the body. As HLHS is often fatal without several surgical procedures, the search for an improvement of its treatment has been of great scientific interest.  

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Lomecel-B is a medicinal signaling cell therapy, currently under investigation as a novel potential treatment for HLSP, to be administered during cardiovascular surgery. Phase I clinical trials of Lomecel-B attempted this treatment alongside surgery and have shown great promise.  All infants involved in the trial survived, showed normal growth pattern and weight gain, and did not require a transplant 2442 months post-surgery. Furthermore, no adverse reactions or infection were reported post-surgery.  

“We are encouraged by our phase I clinical data, and the progress being made in the ongoing phase II 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,” commented Joshua Hare, Chief Science Officer and Co-Founder of Longeveron. “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.” 

RPD designation is granted by the FDA for rare pediatric diseases (<200,000 individuals). Under this designation, Lomecel-B has been approved for the treatment of HLHS and could be eligible to receive a priority review voucher. The receipt of this voucher could potentially accelerate the time for this cell therapy to reach the market to treat this deadly disease.