idursulfase-IT + idursulfase

**idursulfase-IT + idursulfase** is a combination therapy used in the management of *neuronopathic mucopolysaccharidosis II (MPS II, Hunter syndrome)*. **Idursulfase** is a recombinant form of the human lysosomal enzyme *iduronate-2-sulfatase* administered intravenously; it increases lysosomal catabolism of glycosaminoglycans (GAGs, specifically dermatan sulfate and heparan sulfate), treating the somatic symptoms of MPS II[4][5]. **Idursulfase-IT** (investigational code names SHP609/HGT-2310) is a formulation designed for intrathecal administration to deliver enzyme directly to the central nervous system, intended to address neurocognitive decline unresponsive to IV therapy because the normal formulation does not cross the blood-brain barrier[1][2][3]. The combination aims to provide both systemic and central nervous system disease control. Idursulfase-IT has been evaluated in clinical trials combined with weekly IV idursulfase[1][2]. The mechanism of action is lysosomal enzyme replacement to decrease the accumulation of GAGs, thereby reducing pathological tissue storage and cellular dysfunction. Developers include Shire (now part of Takeda). Primary indication: MPS II (Hunter syndrome), especially with neurocognitive impairment[1][2][3].

Other names

iduronate-2-sulfatase (intrathecal and intravenous)recombinant human I2S (intrathecal and intravenous)