Shire Provides Regulatory Progress Update for Key Pipeline Programs Targeting Rare Disease

- U.S. FDA Grants Orphan Drug Designation for Investigational SHP647 for Treatment of Pediatric Patients with Moderately to Severely Active Crohn's Disease

- Health Canada Grants Priority Review for Investigational Lanadelumab for Prevention of Angioedema Attacks in Patients 12 Years and Older with Hereditary Angioedema

Cambridge, MA, USA I February 13, 2018 I Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Shire's investigational therapy, SHP647, for the treatment of pediatric patients with moderately to severely active Crohn's disease. Shire is preparing to conduct Phase 3 trials investigating SHP647 for the treatment of moderately to severely active Crohn's disease in adults. Pediatric study plans with SHP647 are currently under discussion with health authorities.

Health Canada has accepted Shire's request for priority review for the New Drug Submission (NDS) for lanadelumab (SHP643), an investigational therapy for prevention of angioedema attacks in patients 12 years and older with hereditary angioedema (HAE). Health Canada's review of the NDS under Priority Review is expected to be completed in the second half of 2018.

Pediatric onset Crohn's disease presents often with a more complicated disease course compared to adults and can have an impact on patients' growth, pubertal and emotional development.[1] In the U.S., the prevalence of Crohn's disease in the pediatric population is approximately 58 in 100,000.[2]

HAE is a rare, genetic disorder that results in recurring attacks of edema (swelling) in various parts of the body, including the abdomen, face, feet, genitals, hands and throat.[3],[4],[5]
The swelling can be debilitating and painful, resulting in significant impact on quality of life for those individuals with HAE.

U.S. FDA 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.[6]

Priority review status is granted by Health Canada to a NDS for serious, life-threatening or severely debilitating diseases or conditions for which there is substantial evidence of clinical effectiveness that the drug provides an effective treatment, prevention or diagnosis of a disease or condition for which no drug is currently marketed in Canada; or a significant increase in efficacy and/or significant decrease in risk such that the overall benefit/risk profile is improved over existing therapies, preventatives or diagnostic agents for a disease or condition that is not adequately managed by a drug marketed in Canada.[7]

About SHP647
SHP647 is a fully human IgG2 monoclonal antibody targeting the mucosal vascular addressin cell adhesion molecule-1 (MAdCAM-1). 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. Shire is currently investigating SHP647 in Phase 3 studies for the treatment of moderately to severely active ulcerative colitis, a form of inflammatory bowel disease, in adults. Shire is also preparing to conduct Phase 3 trials investigating SHP647 for the treatment of moderately to severely active Crohn's disease in adults. Pediatric study plans with SHP647 are currently under discussion with health authorities. In November 2017, SHP647 received Orphan Drug Designation from the U.S. FDA for the treatment of pediatric patients with moderately to severely active ulcerative colitis.

About Lanadelumab
Lanadelumab is an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein[8] and is being studied as a treatment for the prevention of angioedema attacks in patients 12 years and older with HAE. Lanadelumab is formulated for subcutaneous administration, and has a half-life of approximately 14 days in patients with HAE.[9]

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.

www.shire.com

[1] Ruemmele FM et al J Crohns Colitis. 2014 Oct;8(10):1179-207. doi: 10.1016/j.crohns.2014.04.005.

[2] Kappelman MD  Dig Dis Sci. 2013 Feb;58(2):519-25. doi: 10.1007/s10620-012-2371-5.

[3] Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.

[4] Cicardi M, Bork K, Caballero T, et al, on behalf of HAWK (Hereditary Angioedema International Working Group). Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012;67(2):147-157.

[5] Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.

[6] https://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/OfficeofScienceandHealthCoordination/ucm2018190.htm.

[7] https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/applications-submissions/guidance-documents/priority-review/drug-submissions.html

[8] Kenniston JA et al. Inhibition of plasma kallikrein by a highly specific active site blocking antibody. J. Biol. Chem. 2014;289(34):23596-23608.

[9] Banerji et al. Inhibiting plasma kallikrein for hereditary angioedema prophylaxis. N Engl J Med. 2017; 376(8):717-728.

SOURCE: Shire

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