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New Formulation of ONCASPAR®▼ (pegaspargase) Receives Marketing Authorization in Europe for Patients with Acute Lymphoblastic Leukemia (ALL)


Freeze-dried, or lyophilized, formulation aims to improve supply management
by providing the same dosing regimen as liquid ONCASPAR,
but with a three-times longer shelf life of up to 24 months1

Zug, Switzerland – December 13, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global leader in rare diseases, today announced that the European Commission (EC) granted Marketing Authorization for lyophilized ONCASPAR (pegaspargase), as a component of antineoplastic combination therapy in acute lymphoblastic leukemia (ALL) in pediatric patients from birth to 18 years, and in adult patients.1 The approval – which authorizes Shire to market lyophilized ONCASPAR in the 28 member states of the European Union (EU), as well as Iceland, Liechtenstein and Norway – follows a positive opinion adopted by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on October 12.

“This approval underscores Shire’s commitment to patients with acute lymphoblastic leukemia through continued research and evolution of asparaginase therapy,” said Howard B. Mayer, M.D., SVP and ad-interim Head, Global Research and Development, Shire. “With this lyophilized formulation, we aim to make pegylated asparaginase, part of the pediatric standard therapy in acute lymphoblastic leukemia, available to patients in countries where liquid ONCASPAR is not currently offered. Additionally, with extended shelf life up to 24 months, treatment centers will have flexibility in inventory management to help ensure continuous treatment supply for patients.”

Lyophilized ONCASPAR builds on more than a decade of data and research with liquid ONCASPAR, a pegylated asparaginase, and works the same way as the liquid formulation. By depleting serum L-asparagine levels and thereby interfering with protein synthesis, ONCASPAR deprives lymphoblasts of L-asparagine, resulting in cell death.2,3,4

The new lyophilized formulation offers the same dosing regimen as liquid ONCASPAR, but with a three-times longer shelf life than the liquid formulation.1 Asparaginase is a critical component of the treatment regimen for ALL patients as it is a proven approach to inducing leukemic cell death.2,3,4

Shire expects lyophilized ONCASPAR to be available in European markets beginning in the first half of 2018.

About Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is a cancer of the white blood cells and is characterized by an overproduction and accumulation of lymphoblasts, immature white blood cells. ALL is the most common type (~75%) of cancer among children diagnosed with leukemia5. ALL can be curable within certain pediatric patient populations, with a five-year overall survival rate of 96% in children treated with regimens including ONCASPAR6.

About ONCASPAR
In Europe, ONCASPAR is indicated as a component of antineoplastic combination therapy in acute lymphoblastic leukemia (ALL) in pediatric patients from birth to 18 years, and adult patients.1

Safety Information in Europe
ONCASPAR is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients, in patients with severe hepatic impairment, and in patients with a history of serious thrombosis, pancreatitis, or serious hemorrhagic events with prior L-asparaginase therapy.

Anaphylaxis or serious allergic reactions can occur; therefore, patients should be observed for 1 hour after administration having resuscitation equipment ready. Discontinue ONCASPAR in patients with serious allergic reactions. There have been reports of adverse reactions of pancreatitis. If pancreatitis is suspected ONCASPAR should be discontinued. ONCASPAR should also be discontinued in patients with serious thrombotic events.

Combination therapy with ONCASPAR can result in hepatic toxicity and central nervous system toxicity. In the presence of symptoms of hyperammonemia (e.g. nausea, vomiting, lethargy, irritation), ammonia levels should be monitored closely.

Very common adverse reactions reported in clinical trial data and the post‑marketing experience of ONCASPAR in ALL patients include: hyperglycemia, pancreatitis, diarrhea, abdominal pain, nausea; hypersensitivity, urticaria, anaphylactic reactions; weight decreased; decreased appetite; rash.

Common adverse reactions include: febrile neutropenia, anemia, thrombosis; vomiting, stomatitis; hepatotoxicity, fatty liver; infections, sepsis; amylase increased, alanine aminotransferase increased, blood bilirubin increased, blood albumin decreased, neutrophil count decreased, platelet count decreased, activated partial thromboplastin time prolonged, prothrombin time prolonged, hypofibrinogenemia; hypertriglyceridemia, hyperlipidemia, hypercholesterolemia; pain in extremities; seizure, peripheral motor neuropathy, syncope; hypoxia; thrombosis.1

For further information please contact:

Investor Relations  
Ian Karpikarp@shire.com+1 781 482 9018
Robert Coatesrcoates@shire.com+44 203 549 0874
Media  
Gwen Fishergfisher@shire.com +1 781 482 9649
Annabel Cowperannabel.cowper@shire.com+41 44 878 6638

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.

www.shire.com

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a further list and description of risks, uncertainties and other matters can be found in Shire’s most recent Annual Report on Form 10-K and in Shire’s subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in “ITEM 1A: Risk Factors”, and in Shire’s subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire’s website.

All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to update or revise forward-looking statements, whether as a result of new information, future events or otherwise.

1 Lyophilized Oncaspar Summary of Product Characteristics. European Medicines Agency.
2 Oncaspar (pegaspargase) Summary of Product Characteristics. (SmPC) EU 1/2016.
3 Wilson GJ, Bunpo P, Cundiff JK, et al. The eukaryotic initiation factor 2 kinase GCN2 protects against hepatotoxicity during asparaginase treatment. Am J Physiol Endocrinol Metab. 2013;305:E1124–1133.
4 Story MD, Voehringer DW, Stephens LC, et al. L-Asparagainse kills lymphoma cells by apoptosis. Cancer Chemother Pharmacol. 1993;32:129–133.
5 What is Childhood Leukemia? American Cancer Society. Available at: https://www.cancer.org/cancer/leukemia-in-children/about/what-is-childhood-leukemia.html. Accessed November 21, 2017.
6 Place AE, Stevenson KE, Vrooman LM, et. al. Intravenous pegylated asparaginase versus intramuscular native Escherichia coli L-asparaginase in newly diagnosed childhood acute lymphoblastic leukaemia (DFCI 05-001): a randomised, open-label phase 3 trial. Lancet Oncol. 2015;16:1677-90.

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