Lomecel-B (Longeveron) is an experimental allogeneic bone marrow-derived signaling cell medicinal drug that is being evaluated in a Phase 2 trial.
The FDA has awarded Lomecel-B (Longeveron) the rare childhood disease (RPD) designation 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 to these severely affected infants at the same time as surgery,” Joshua Hare, MD, chief science officer at Longeveron, said in the statement.
Lomecel-B is an experimental allogeneic bone marrow-derived signaling cell medicinal drug that is being evaluated in a Phase 2 trial.
In the Phase 1 clinical trial, the company reported that when cardiac surgeons injected the medication into the baby’s hearts at the time of surgery, the cells were well tolerated with no major adverse cardiac events. There were no infections related to treatment.
The phase 1 study showed that all babies survived and did not need a transplant between 2 and 3 and a half years after surgery. The growth pattern and weight gain were consistent with normal infant measurements.
HLHS is an underdevelopment of the left ventricle of the heart, which affects the heart’s ability to pump blood around the body and is often fatal without a series of surgeries.
The FDA grants RPD designation for diseases that primarily affect individuals 18 years of age or younger and fewer than 200,000 individuals in the United States.
If the FDA approves Lomecel-B for treatment, Longeveron may be eligible for a priority review voucher if the application is made for the product that meets certain conditions and current legislation.
The US Food and Drug Administration approves Longeveron’s Lomecel-B for the designation of rare childhood disease for the treatment of life-threatening heart disease in infants. EurekAlert. Globe Newswire. E-mail. November 18, 2021. Accessed November 18, 2021.