FDA Fast-Tracks Investigational Hunter Syndrome Formulation

Shire recently announced that the FDA has granted Fast Track designation to its investigational treatment for neurocognitive decline associated with Hunter syndrome (MPS II).

Idursulfase-IT is a formulation of Shire’s currently approved Hunter syndrome treatment, idursulfase (Elaprase), that can be directly administered into the cerebrospinal fluid via an intrathecal drug delivery device. In contrast with the new formulation, Elaprase is administered intravenously and does not cross the blood-brain barrier in sufficient amounts.

The FDA’s inclusion of idursulfase-IT in its Fast Track program will expedite the drug’s review process and increase its likelihood of being eligible for priority review if Shire can provide ample clinical data.

“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 Philip J. Vickers, MD, head of research and development at Shire, in a press release. “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.”

Shire is currently enrolling patients in a controlled, randomized, open-label, multi-center, assessor-blinded phase 2/3 trial to assess the effect of idursulfase IT in pediatric Hunter syndrome patients with early cognitive impairment who receive and tolerate Elaprase. The manufacturer is also planning an extension study to evaluate the drug’s long-term safety and efficacy.