FDA Grants Rare Pediatric Disease Designation for Treatment of HLHS in Infants

The FDA has granted rare pediatric disease (RPD) designation to Lomecel-B (Longeveron) for the treatment of hypoplastic left heart syndrome (HLHS), a life-threatening and rare congenital heart defect in infants, Longeveron said in a statement.

“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,” Joshua Hare, MD, chief science officer at Longeveron, said in the statement.

Lomecel-B is an investigational allogeneic, bone marrow-derived medicinal signaling cell medication that is being evaluated in a phase 2 trial.

In the phase 1 clinical study, the company reported that when cardiac surgeons injected the medication into the infant’s hearts at the time of surgery, the cells were well tolerated with no major adverse cardiac events. No infections were related to the treatment.

The phase 1 trial showed that all the infants survived and had not required a transplant between 2 to 3 and a half years, post-surgery. Growth pattern and weight gain matched the normal measurements of infants.

HLHS is underdevelopment of the left ventricle, which impairs the heart’s ability to pump blood throughout the body and is often fatal without a series of surgeries.

The FDA grants RPD designation for diseases that primarily affect individuals aged 18 years or younger and fewer than 200,000 individuals in the United States.

If the FDA approves Lomecel-B for treatment, Longeveron may be eligible to receive a priority review voucher if the applicating is submitted for the product satisfied under certain conditions and current law.

Reference

U.S. Food and Drug Administration approves Longeveron’s Lomecel-B for rare pediatric disease designation to treat life-threatening infant heart condition. EurekAlert. Globe Newswire. Email. November 18, 2021. Accessed November 18, 2021.