Shire’s good run is continuing with the news that the US Food and Drug Administration has granted fast-track designation for SHP609 (idursulfase-IT) for the treatment of neurocognitive decline associated with Hunter syndrome.
The treatment is a new formulation of Shire’s already-approved Hunter syndrome drug Elaprase (idursulfase) which has been designed for direct administration into the cerebrospinal fluid via an intrathecal drug delivery device. The syndrome, also known as mucopolysaccharidosis II (MPS II), is a rare disease that affects one in 162,000 total live births and is caused by a deficiency or absence of the lysosomal enzyme iduronate-2-sulfatase (I2S).
Shire R&D chief Philip Vickers noted that “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 programme for enzyme replacement has ever progressed”. He added that getting fast-track designation “is further recognition of the critical need to develop new, effective therapy options for patients with Hunter syndrome”.
On Friday, the FDA approved Natpara (recombinant human parathyroid hormone) for hypoparathyroidism from NPS Pharmaceuticals, which is in the process of being acquired by Shire.
