US regulators have granted Shire’s investigational therapy SHP647 orphan status for the treatment of pediatric patients with moderately to severely active ulcerative colitis (UC).
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 that targets the mucosal addressin cell adhesion molecule-1 (MAdCAM-1), which plays a role in leukocyte migration in the GI tract and also appears to facilitate excessive lymphocyte infiltration in the presence of chronic GI inflammation.
The drug is currently being trialed by the company in Phase III studies for the treatment of moderately to severely active UC in adults.
“If approved, SHP647 holds the potential to help treat patients with ulcerative colitis,” noted Debra Silberg, therapeutic area development lead for GI, Endocrine, and Metabolism at Shire.
She also noted that the firm’s plans to test the drug in the paediatric population, which are currently being discussed with health authorities, “align well with our commitment to address unmet patient need.”
Shire licensed SHP647 from Pfizer in June last year.