MPS: In The News

Orphan Druganaut Blog

This Blog Post will review recent developments for the rare disease Mucopolysaccharidosis (MPS).

I – Shire’s FDA Fast Track Designation For MPS II (Hunter Syndrome)

Shire announces January 26th that the company’s investigational product, SHP609 (Idursulfase-IT), is granted FDA Fast Track designation for the neurocognitive decline associated with MPS II (Hunter Syndrome). The product receives FDA Orphan Drug Designation (ODD) in September 2009. SHP609 is for direct administration into the cerebrospinal fluid via an intrathecal drug delivery device (IDDD) and is being developed for use with Shire’s drug Elaprase (Idursulfase), which is FDA approved for Hunter Syndrome. Elaprase is intravenously administered (ERT) and doesn’t cross the blood-brain barrier in any relevant amounts.

Hunter Syndrome is a progressive, rare, and severe Lysosomal Storage Disorder (LSD), caused by inadequate activity of the enzyme that breaks down complex sugars – Iduronate-2-Sulfatase (IDS).

A Shire Phase II/III pivotal trial (HGT-HIT-094 or AIM-IT

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