Posted by Michael Wonder on 07 May 2024
iECURE receives FDA fast track designation for ECUR-506 for the treatment of neonatal onset ornithine transcarbamylase deficiency
6 May 2024 - iECURE announced today that it has received fast track designation from the US FDA for ECUR-506, the company’s in vivo gene insertion program designed to treat neonatal onset ornithine transcarbamylase deficiency.
ECUR-506 previously received rare paediatric disease and orphan drug designations from the FDA, and orphan designation from the European Commission for the treatment of ornithine transcarbamylase deficiency.
