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Shire launches myPKFiT™ for ADVATE® [Antihemophilic Factor (Recombinant)] in the U.S.

First and only FDA-cleared PK dosing software now available to help healthcare professionals create personalized
treatment regimens for patients 16 and older with hemophilia A1

Cambridge, Mass. – March 5, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, today announced the U.S. availability of myPKFiT for ADVATE [Antihemophilic Factor (Recombinant)], a free web-based, Rx software that is the first and only pharmacokinetic (PK) dosing software cleared by the U.S. Food and Drug Administration (FDA) for use with hemophilia A patients 16 and older weighing at least 45 kilograms and treated with ADVATE.1 The software generates a patient’s estimated individual PK profile, which aids healthcare professionals in personalizing a patient’s prophylaxis ADVATE dose and schedule.1

“A version of myPKFiT has already been widely adopted in Europe since 2014, and we’ve seen how important it has been in helping physicians develop personalized dosing regimens tailored to the specific needs of their patients,” said Andreas Busch, Global Head of Research and Development, Shire. “As part of our commitment to precision medicine, we are pleased to bring this innovative application to physicians and patients in the United States.”

The myPKFiT for ADVATE software offers an innovative approach to estimating a patient’s PK curve, a key measure for assessing drug exposure over time.1,2 With myPKFiT for ADVATE, healthcare professionals can now estimate a full PK curve with as few as two measurable blood samples, compared to the 9 to 11 recommended by the International Society on Thrombosis and Haemostasis (ISTH) guidelines.1,3

“We understand that people with hemophilia have their own treatment goals, needs and challenges that require a customized approach to care,” said Michael Tarantino, M.D., Medical Director and President, The Bleeding and Clotting Disorders Institute. “The myPKFiT software enables healthcare professionals to gain an even clearer picture of their patients’ treatment needs to develop an ADVATE prophylaxis dose and schedule that’s truly tailored to each individual patient.”

Hemophilia A, the most common type of hemophilia, is a rare bleeding disorder that causes longer-than-normal bleeding due to a lack of clotting factor VIII (FVIII) in the blood.4 The severity of hemophilia A is determined by the amount of factor in the blood, with more severity associated with lower amounts of factor.5 More than half of patients with hemophilia A have the severe form of the condition.5 Hemophilia primarily affects males, with an incidence of one in 5,000 male births in the United States.6

myPKFiT for ADVATE software is Rx Only. For safe and proper use of the myPKFiT for ADVATE software, please refer to the complete instructions for use in the User Manual.

myPKFiT for ADVATE Indications for Use8

The myPKFiT software is intended for use by licensed healthcare professionals (HCPs) who are familiar with hemophilia care. The myPKFiT software can be used to generate ADVATE dosage amount and frequency recommendations for routine prophylaxis for an individual patient 16 years of age and older and body weight of 45 kg or greater, using that patient’s age and body weight information and local laboratory FVIII one-stage clotting activity measurements of sparse samples collected from that patient.

A minimum of two sparse sampling points are required at the recommended 3-4 hours (± 30 minutes) and at 24-32 hours (±1 hour) post-infusion.

HCPs will also be able to evaluate various prophylaxis dose regimens tailored to an individual patient’s needs and treatment plan.

The software output may be used to guide decisions on appropriate ADVATE dose and infusion intervals to maintain FVIII activity levels at or above a user specified minimum FVIII activity level between 1% to 3% above natural baseline for an individual patient in accordance with the FDA-approved dosing recommendations provided in the ADVATE Prescribing Information (PI).

myPKFiT should only be used to evaluate prophylactic dosing regimens for hemophilia A patients treated with ADVATE, as per the ADVATE Prescribing Information (PI).

myPKFiT for ADVATE is not indicated for the treatment of von Willebrand disease. myPKFiT for ADVATE should not be used for patients who have developed neutralizing antibody (inhibitor) to FVIII products.

About ADVATE
ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives.9

ADVATE is currently approved in 69 countries worldwide, including the United States, Canada, 28 countries in the European Union, Algeria, Argentina, Australia, Brazil, Brunei, Chile, China, Colombia, Ecuador, Hong Kong, Iceland, India, Iraq, Israel, Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New Zealand, Norway, Panama, Puerto Rico, Qatar, Russia, Saudi Arabia, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.

ADVATE [Antihemophilic Factor (Recombinant)] Important Information

Indications
ADVATE is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency) for:
• Control and prevention of bleeding episodes.
• Perioperative management.
• Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

ADVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS
Patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.

WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with ADVATE. Symptoms include dizziness, paresthesia, rash, flushing, facial swelling, urticaria, dyspnea, pruritus, and vomiting. Discontinue ADVATE if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Neutralizing Antibodies
Neutralizing antibodies (inhibitors) have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs). Monitor all patients for the development of factor VIII inhibitors by appropriate clinical observation and laboratory testing. If expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.

ADVERSE REACTIONS

  • Serious adverse reactions seen with ADVATE are hypersensitivity reactions, including anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.
  • The most common adverse reactions observed in clinical trials (>5% of subjects) were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea.

Please see accompanying ADVATE full Prescribing Information available at http://www.shirecontent.com/PI/PDFs/ADVATE_USA_ENG.pdf

For more information on ADVATE, please visit www.ADVATE.com.

For further information please contact:

Media Relations
Elizabeth Kalinaekalina@shire.com+1 781 482 2713
Gwen Fishergfisher@shire.com+1 781 482 9649
Investor Relations
Christoph Brackmannchristoph.brackmann@shire.com+41 795 432 359
Sun Kimsun.kim@shire.com+1 617 588 8175
Robert Coatesrcoates@shire.com+44 203 549 0874

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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Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, projected revenues, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products, are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:

  • Shire’s products may not be a commercial success;
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  • the nature of producing plasma-based therapies may prevent Shire from timely responding to market forces and effectively managing its production capacity;
  • Shire has a portfolio of products in various stages of research and development. The successful development of these products is highly uncertain and requires significant expenditures and time, and there is no guarantee that these products will receive regulatory approval;
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  • Shire’s products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
  • Shire’s patented products are subject to significant competition from generics;
  • adverse outcomes in legal matters, tax audits and other disputes, including Shire’s ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the Shire’s revenues, financial condition or results of operations;
  • Shire may fail to obtain, maintain, enforce or defend the intellectual property rights required to conduct its business;
  • Shire faces intense competition for highly qualified personnel from other companies and organizations;
  • failure to successfully execute or attain strategic objectives from Shire’s acquisitions and growth strategy may adversely affect the Shire’s financial condition and results of operations;
  • Shire’s growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
  • a slowdown of global economic growth, or economic instability of countries in which Shire does business, could have negative consequences for Shire’s business and increase the risk of non-payment by Shire’s customers;
  • changes in foreign currency exchange rates and interest rates could have a material adverse effect on Shire’s operating results and liquidity;
  • Shire is subject to evolving and complex tax laws, which may result in additional liabilities that may adversely affect the Shire’s financial condition or results of operations;
  • if a marketed product fails to work effectively or causes adverse side effects, this could result in damage to Shire’s reputation, the withdrawal of the product and legal action against Shire;
  • Shire is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on Shire’s revenues, financial condition or results of operations;
  • Shire faces risks relating to the expected exit of the United Kingdom from the European Union;
  • Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which has increased its borrowing costs and may decrease its business flexibility;
  • Shire's ongoing strategic review of its Neuroscience franchise may distract management and employees and may not lead to improved operating performance or financial results; there can be no guarantee that, once completed, Shire's strategic review will result in any additional strategic changes beyond those that have already been announced; and

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 “ITEM1A: 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.

References:

1 U.S. Food and Drug Administration, Center for Biologic Evaluation and Research Office of Tissues and Advanced Therapies. myPKFiT for ADVATE Approval Letter BK170028, December 18, 2017.
2 Gustafson DL and Bradshaw-Pierce EL. Fundamental Concepts in Clinical Pharmacology. M. Hidalgo et al. (eds.), Principles of Anticancer Drug Development, Cancer Drug Discovery and Development, DOI 10.1007/978-1-4419-7358-0_2. Accessible at: http://media.axon.es/pdf/83236_2.pdf
3 Lee M et al. “Scientific and Standardization Committee Communication The Design and Analysis of Pharmacokinetic Studies of Coagulation Factors.” ISTH Website. https://c.ymcdn.com/sites/www.isth.org/resource/group/d4a6f49a-f4ec-450f-9e0f-7be9f0c2ab2e/official_communications/fviiipharmaco.pdf.
4 World Federation of Hemophilia. “What is hemophilia?” World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=646.
5 National Hemophilia Foundation. “Hemophilia A.” National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A.
6 Centers for Disease Control and Prevention. “Hemophilia Data & Statistics”. CDC website. https://www.cdc.gov/ncbddd/hemophilia/data.html.
7 Securities and Exchange Commission. “General Form for Registration of Securities, Baxalta Incorporated”. 2014 Accessible at: https://www.sec.gov/Archives/edgar/data/1620546/000119312516431188/d76818ds1a.htm
8 myPKFiT Device User Manual. Baxter Healthcare Corporation; 2015.
9 ADVATE Prescribing Information.

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