U.S. FDA Grants Shire Orphan Drug Designation for Investigational Anti-MAdCAM Antibody SHP647 for the Treatment of Moderately to Severely Active Ulcerative Colitis in Pediatric Patients

U.S. FDA Grants Shire Orphan Drug Designation for Investigational Anti-MAdCAM Antibody SHP647 for the Treatment of Moderately to Severely Active Ulcerative Colitis in Pediatric Patients

Cambridge, Ma.  - November 30, 2017 - Shire plc (LSE: SHP, NASDAQ: SHPG), the world's leading biotechnology company in rare diseases, today announced that the U.S. Food and Drug Administration (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). SHP647 is a fully human IgG2 monoclonal antibody targeting the mucosal addressin cell adhesion molecule-1 (MAdCAM-1). 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.

Orphan Drug status is intended to advance drug development for rare diseases.The FDA provides Orphan Drug Designation to drugs and biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S. UC is a chronic, relapsing and remitting inflammatory disorder of the colon. 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. Symptoms can be debilitating and include bloody diarrhea, tenesmus, abdominal pain, and in severe cases, weight loss, fatigue, and vomiting.

Debra Silberg, M.D., Ph.D., Therapeutic Area Development Lead for GI, Endocrine, and Metabolism at Shire, remarked, "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. 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 alpha₄beta₇ integrin binding to human MAdCAM-1 with high affinity and selectivity.

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NOTES TO EDITORS

About Shire

Shire is the global leader in serving patients with rare diseases. We strive to develop best-in-class therapies across a core of rare disease areas including hematology, immunology, genetic diseases, neuroscience, and internal medicine with growing therapeutic areas in ophthalmics and oncology. Our diversified capabilities enable us to reach patients in more than 100 countries who are struggling to live their lives to the fullest.

We feel a strong sense of urgency to address unmet medical needs and work tirelessly to improve people's lives with medicines that have a meaningful impact on patients and all who support them on their journey.

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