Rare Disease Report

Intrathecal ERT to Treat Cognitive Decline in Hunter Syndrome Gets Fast Tracked

JANUARY 25, 2015
James Radke

An intrathecal enzyme replacement therapy (idursulfase-IT) has been granted Fast Track designation by the United States Food and Drug Administration (FDA) to treat the cognitive decline in patients with Hunter syndrome. An intravenous version of idursulfase (Elaprase) is currently approved to treat the peripheral aspects of the disease.

Hunter syndrome is an X-linked lysosomal disorder caused by a deficiency or absence of iduronate-2-sulfatase, which leads to severe clinical complications and early mortality. Common symptoms may include stunted growth, coarse facial features, hearing loss, joint stiffness, sleep apnea, thickening of heart valves, obstructive airways, enlarged liver and spleen, and cognitive impairment.

Philip J. Vickers, PhD, Head of Research and Development at Shire said, "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."

Shire is currently enrolling patients in a Phase II/III pivotal trial (HGT-HIT-094 or AIM-IT), which is a controlled, randomized, open-label, multi-center, assessor-blinded study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly administration of idursulfase IT in pediatric patients with Hunter syndrome and early cognitive impairment who already receive and tolerate therapy with intravenous idursulfase.

For further information on the idursulfase IT pivotal trial, visit https://clinicaltrials.gov/ct2/show/NCT02055118

For more information about Hunter syndrome, visit http://www.savingcase.com/ or http://mpssociety.org/mps/mps-ii/ . To see a video of Saving Cases’s founder Melissa Hogan discuss the disease,  click here.

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