Human heterologous liver cells

Unassigned

New Medicines

Heparesc · Urea cycle disorders in children

Information

Heparesc
Advanced therapy medicinal product (ATMP)
Promethera Biosciences
Promethera Biosciences

Development and Regulatory status

None
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes

Category

Cryopreserved mature human hepatocytes from donor livers, given by portal vein infusion over six days.
Ornithine-transcarbamylase deficiency (Crigler-Najjar syndrome) affects < 0.1 in 10,000 in EU (<5,000 people) and carbamoyl-phosphate synthase-1 deficiency <0.02 in 10,000 (< 1,000 people). The mortality rate >50% at 10 years old and neurological sequelae are common in surviving patients. Orthotopic liver transplantation has long-term survival rates of about 90%. AIM of HHLivC therapy is to compensate for the metabolic defect and reduce the risk of neurological deterioration while awaiting OLT.
Urea cycle disorders in children
Intravenous infusion

Evidence based evaluations