Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Longeveron’s Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome, a rare and life-threatening congenital heart defect in infants.

Approximately 1,000 babies are born with Hypoplastic Left Heart Syndrome (HLHS) each year in the United States. HLHS babies have an underdeveloped left ventricle, which impairs the heart’s ability to pump blood throughout the body. HLHS is often fatal without surgical intervention, in which three surgical procedures are performed over a period of about 5 years, to allow the right ventricle to be configured to pump blood to the body.

Lomecel-B is an investigational allogeneic, bone marrow-derived medicinal signaling cell (MSC) product. Longeveron is evaluating the safety of Lomecel-B injection into the right ventricle during the second surgery (4 to 6 months of age), and the effect on cardiac function and other health status endpoints.

“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,” said Joshua Hare, co-founder and chief science officer of Longeveron. “We are encouraged by our phase 1 clinical data, and the progress being made in the ongoing phase 2 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.”

Longeveron recently reported clinical results from its safety-focused phase I clinical study of Lomecel-B in HLHS patients. When cardiac surgeons injected Lomecel-B directly into the babies’ hearts at the time of surgery, the cells were well tolerated with no major adverse cardiac events and no infections considered to be related to the investigational treatment.

One hundred percent of infants enrolled in the phase 1 trial were alive and had not required a transplant between 2 to 3.5 years post-surgery. Other measurements of the babies’ health, such as weight gain and growth pattern, matched that of normal healthy babies.

Infants with HLHS are currently being enrolled in ELPIS II, a 38-subject, phase 2 randomized, double-blind, controlled clinical trial that is being funded in part by a grant from the National Institute of Health’s National Heart, Lung, and Blood Institute.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes Lomecel-B eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Mirum Pharmaceuticals sold its voucher for $110 million.

Photo: Joshua Hare, co-founder and chief science officer of Longeveron

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