US regulators have awarded orphan drug status to Shire's investigational therapy, SHP647, for the treatment of paediatric patients with moderately to severely active Crohn's disease.
The move qualifies Shire for access to various incentives designed to accelerate development of drugs for rare diseases, including tax credits for qualified clinical testing.
SHP647 is a fully human IgG2 monoclonal antibody targeting the mucosal vascular addressing cell adhesion molecule-1 (MAdCAM-1), which plays a role in leukocyte trafficking in the GI tract and also appears to facilitate excessive lymphocyte infiltration under conditions of chronic GI inflammation.
The firm is already assessing the drug in Phase III studies for the treatment of moderately to severely active ulcerative colitis in adults, for which it also carries an orphan drug designation.
Preparations for Phase III trials investigating the potential SHP647 in adults with severely active Crohn’s disease are already underway, while paediatric study plans are currently under discussion with health authorities.
Shire licensed SHP647 from Pfizer in June 2016.