Date: 2017-11-16
Type of information: Granting of the orphan status in the US
Product name: SHP647 (PF-00547659)
Compound: human IgG2 anti-monoclonal antibody directed against mucosal addressin cell adhesion molecule (MAdCAM)
Therapeutic area: Autoimmune diseases - Inflammatory diseases - Digestive diseases - Rare diseases
Action mechanism:
- monoclonal antibody. SHP647/PF-00547659 is a fully-human monoclonal antibody that is designed to directly target a gastrointestinal endothelial adhesion molecule known as mucosal addressin cell adhesion molecule 1 (MAdCAM-1), that binds to the alpha4beta7 integrin on lymphocytes. The interaction between endothelial MAdCAM-1 and ?4?7 integrin has been implicated in the pathogenesis of ulcerative colitis.3 SHP647 directly targets MAdCAM-1, thereby blocking tissue homing of activated ?4?7 + leukocytes.
- Shire licensed the global rights to all indications for PF-00547659 (SHP647) from Pfizer in June 2016.
Company: Shire (UK - USA)
Disease:
- treatment of pediatric patients with moderately to severely active ulcerative colitis (UC)
Latest news:
- • On November 16, 2017, the FDA granted Orphan Drug Designation to Shire’s investigational anti-MAdCAM-1 antibody, SHP647 (formerly known as PF-00547659), for the treatment of pediatric patients with moderately to severely active ulcerative colitis (UC). In the U.S., the reported incidence of UC in children aged 0-19 years varies between 0.34 and 2.9 per 100,000 children.
- Shire is currently investigating SHP647 in Phase 3 studies for the treatment of moderately to severely active UC in adults. Pediatric study plans with SHP647 are currently under discussion with health authorities.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2017-11-16
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
