Shire plc announces that the United States Food and Drug Administration (FDA) has granted Fast Track designation for SHP609 (idursulfase-IT; also known as HGT-2310) for the treatment of neurocognitive decline associated with Hunter syndrome (mucopolysaccharidosis II or MPS II). This investigational formulation of idursulfase has been designed for direct administration into the cerebrospinal fluid via an intrathecal drug delivery device (IDDD). This formulation is being investigated and developed for use with Shire’s currently approved treatment for Hunter syndrome, Elaprase (idursulfase). Elaprase is administered intravenously and does not cross the blood-brain barrier in clinically relevant amounts.

"This is not only the first treatment being investigated to address the significant unmet need of slowing the cognitive decline in MPS II patients, but also the furthest an intrathecal program for enzyme replacement has ever progressed,” said Dr. Philip J. Vickers, Head of Research and Development at Shire. "This Fast Track designation is further recognition of the critical need to develop new, effective therapy options for patients with Hunter syndrome with cognitive impairment."

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