idursulfase-IT

Idursulfase-IT (SHP609) is an investigational recombinant human iduronate-2-sulfatase (I2S) enzyme replacement therapy developed by Shire (now part of Takeda) for the treatment of pediatric patients with Hunter syndrome (Mucopolysaccharidosis II or MPS II) who exhibit cognitive impairment. While the intravenous formulation of idursulfase (Elaprase) effectively treats systemic manifestations, it does not cross the blood-brain barrier in therapeutic concentrations. Idursulfase-IT is a revised formulation specifically designed for intrathecal administration into the cerebrospinal fluid via an implanted drug delivery device, allowing the enzyme to bypass the blood-brain barrier and directly address the accumulation of glycosaminoglycans (GAGs) in the central nervous system. The drug was evaluated in the Phase 2/3 AIM-IT study (NCT02055118), which failed to meet its primary and key secondary endpoints for improving neurodevelopmental status.

Other names

intrathecal idursulfaseidursulfase IT