Shire gets orphan drug status for paediatric UC antibody
Updated : 12:46
Shire's SHP647 monoclonal antibody has been granted 'orphan drug' status for the treatment of children with moderately to severely ulcerative colitis.
The FTSE 100 rare disease specialist was informed by the US Food & Drug Administration that the investigational antibody had been given the special designation that is intended to aid drug development for rare diseases.
SHP647 is a fully human IgG2 monoclonal antibody targeting the mucosal addressin cell adhesion molecule-1 (MAdCAM-1).
Shire, which is currently investigating SHP647 in Phase 3 studies for the treatment of moderately to severely active UC in adults, said it was discussing pediatric study plans for the antibody with health authorities.
The FDA provides orphan drug status to treatments that demonstrate promise in treating rare diseases or conditions that affect fewer than 200,000 people in the US.
Ulcerative colitis is a chronic, relapsing and remitting inflammatory disorder of the colon, with reported incidence in US children aged 0-19 years varying between 0.34 and 2.9 per 100,000. Symptoms can be debilitating and include bloody diarrhea, tenesmus, abdominal pain, and in severe cases, weight loss, fatigue, and vomiting.
"We’re pleased to receive orphan drug designation for SHP647, and we’re excited about our continued work to develop this compound. If approved, SHP647 holds the potential to help treat patients with ulcerative colitis," said Debra Silberg, Shire's therapeutic area development lead for GI, Endocrine, and Metabolism.
Shire’s study plans for SHP647 in the pediatric population align well with our commitment to address unmet patient need.”
Shire licensed SHP647 from Pfizer in June 2016, adding to Shire’s established and leading gastrointestinal (GI) portfolio. MAdCAM-1 plays a role in leukocyte trafficking in the GI tract and also appears to facilitate excessive lymphocyte infiltration under conditions of chronic GI inflammation. SHP647 directly targets MAdCAM-1, and inhibits α4β7 integrin binding to human MAdCAM-1 with high affinity and selectivity.