New Data From Shire Aim To Help Close the Diagnosis and Treatment Gap for People With Hemophilia

Zug, Switzerland (ots/PRNewswire) -

Shire presentations at EAHAD 2017 underscore the company's
leadership in improving global standards of care

Shire plc (LSE: SHP, NASDAQ: SHPG), the leading biotechnology
company focused on serving individuals with rare diseases, presented
its new estimate of the Global Annual Bleed Rate (GABR), an effort to
build on current global metrics to measure the global burden of
hemophilia.[1] The GABR aims to identify regions of the world with
the greatest need for improved diagnosis and access to approved
treatment options for hemophilia patients.[1] This initial
presentation of the GABR model is one of 12 presentations from Shire
at the 10th Annual Congress of the European Association for
Haemophilia and Allied Disorders (EAHAD), February 1-3, 2017 in
Paris, France.

Initial findings from the GABR research presented at EAHAD show
the incidence of hemophilia may be more than three times current
global estimates and a bleeding episode occurs every three to 15
seconds worldwide.[1] This finding builds on existing data showing
only 25 percent of people living with hemophilia receive adequate
treatment.[2] The comprehensive findings from the model, which will
be published in 2017, aim to help highlight the significant unmet
needs that remain while expanding and improving strategies for
hemophilia care around the world. (Estimating the Global Annual Bleed
Rate in Haemophilia, http://onlinelibrary.wiley.com/doi/10.1111/hae.1
3150/full#hae13150-sec-0190.)[1]

"We know thousands of hemophilia patients continue to bleed
regularly, particularly in the most undertreated regions of the
world, which impacts their joint health, quality of life and ability
to work or go to school," said Alok Srivastava, MD, co-author of the
GABR research and professor of medicine at the Christian Medical
College, Vellore in India. "We must continue to invest in data
collection, particularly patient reported outcomes such as ABR, to
understand the true patient experience and drive important
conversations about improving standards of care globally."

"Hemophilia patients across the world have complex needs and
treatment goals that cannot be met with a one-size-fits-all
approach," said Leonard Valentino, M.D., Global Head of Hematology
Medical Affairs, Shire. "As the global leader in rare hematology,
Shire is committed to innovative research on behalf of this community
to help us focus efforts on improving care for patients."

Additional Shire Data at EAHAD

Shire also presented data at EAHAD confirming the safety and
efficacy profile of its factor replacement portfolio of treatments
for patients with hemophilia. Notably, Shire presented new data from
an analysis of a sequence identical analogue of one investigational
agent, emicizumab, in combination with approved bypassing agents,
FEIBA® [Anti-Inhibitor Coagulant Complex] and recombinant (r)FVIIa,
both treatments for hemophilia patients who develop an inhibitor.[3]
Results found a multi-fold increase in thrombin generation,
indicating a potential thrombotic risk, for patients who receive the
investigational agent combined with FEIBA or rFVIIa. (Synergistic
Effects of a Procoagulant Bispecific Antibody and FEIBA or Factor
VIIa on Thrombin Generation, http://onlinelibrary.wiley.com/doi/10.11
11/hae.13158/full#hae13158-sec-0058.) [3]

In addition, Shire presented an update on its safety database
describing 40 years of real-world safety experience with the
bypassing agent FEIBA.[4] A global review of safety databases for
adverse event reports of FEIBA received from 1975 through July 2016
confirms the safety profile of FEIBA, showing that the reporting rate
of treatment-associated thrombo-embolic events (TEEs) associated with
FEIBA is comparable with previously reported data. (Four Decade
Cumulative Review of Thrombo-Embolic Events Reported with the Use of
Activated Prothrombin Complex Concentrate in Congenital Haemophilia,
http://onlinelibrary.wiley.com/doi/10.1111/hae.13150/full#hae13150-se
c-0012.)[4]

About Hemophilia

Hemophilia is a challenging chronic disease that causes
longer-than-normal bleeding due to absent or deficient clotting
factor in the blood.[5] Hemophilia A is more common than hemophilia
B;[5] hemophilia A affects about 150,000 people, whereas hemophilia B
affects about 30,000 people worldwide.[6]

People with hemophilia, working closely with their healthcare
professionals, can live healthy lives with proper care and adequate
treatment.[2] Treatment regimens typically include on-demand and/or
regular prophylactic infusions of factor replacement therapy to
control or prevent the risk of bleeding.[5],[7]

Inhibitors are a rare but serious complication in patients with
hemophilia.[8] They form when the body's immune system attacks the
molecules in factor therapy, causing it to be ineffective.[8]
Bypassing agents help bypass the inhibitor to help the body form a
clot and stop bleeding.[8]

About the Global Annual Bleed Rate (GABR) Model

In collaboration with renowned hemophilia experts, Shire estimated
the GABR to create a more comprehensive view of the burden of
bleeding on hemophilia patients worldwide.[1] By evaluating
currently-available hemophilia data on prevalence and level of
treatment, a literature-based model was developed to estimate the
GABR at both the country and global levels. A full report of the GABR
model is expected to be published later in 2017.[1]

Safety Statement

Please consult the FEIBA Summary Product Characteristics (SPC)
before prescribing, particularly in relation to dosing and treatment
monitoring.

Contraindications are hypersensitivity to the product,
disseminated intravascular coagulation (DIC) and acute thrombosis or
embolism (including myocardial infarction).

The Adverse Drug Reactions (ADRs) occurring in the highest
frequency (common, >= 1/100 to <1/10) were hypersensitivity,
headache, dizziness, hypotension, rash, hepatitis B surface antibody
positive.

Other symptoms of hypersensitivity reactions to plasma-derived
products include lethargy and restlessness.

Detailed Safety Statement

Please consult the FEIBA Summary Product Characteristics (SPC)
before prescribing, particularly in relation to dosing and treatment
monitoring.

Contraindications

Hypersensitivity to the product or any of the components,
disseminated intravascular coagulation (DIC) and acute thrombosis or
embolism (including myocardial infarction).

Special warnings and precautions for use

Thromboembolic events, including disseminated intravascular
coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial
infarction, and stroke, have occurred in the course of treatment with
FEIBA. At the first signs or symptoms of thromboembolic events, the
infusion should be stopped immediately and appropriate diagnostic and
therapeutic measures initiated.

FEIBA can precipitate allergic-type hypersensitivity reactions;
these reactions can be severe and can be systemic (e.g., anaphylaxis
with urticaria and angioedema, bronchospasm, and circulatory shock).
Other infusion reactions, such as chills, pyrexia, and hypertension
have also been reported.

If clinically significant changes in blood pressure or pulse rate,
respiratory distress, coughing or chest pain occur, the infusion is
to be discontinued immediately and appropriate diagnostic and
therapeutic measures are to be initiated.

In vitro tests, such as aPTT, whole blood clotting time (WBCT) may
not correlate with the clinical picture.

Appropriate vaccination (hepatitis A and B) should be considered
for patients in regular/ repeated receipt of human plasma-derived
products including FEIBA.

Adverse Reactions

Common

(greater than or
equal to1/100 to Hypersensitivity, Headache, Hypotension, Rash,
Hepatitis B
<1/10) surface antibody positive.
Not known Disseminated intravascular coagulation (DIC),
Increase of
inhibitor titer (anamnestic
response),Urticaria, Anaphylactic
reaction, Paresthesia, Hypoaesthesia,
Thrombotic stroke,
Embolic stroke, Somnolence, Dizziness,
Dysgeusia, Cardiac
infarction, Tachycardia, Thrombosis, Venous
thrombosis,
Arterial thrombosis, Embolism, Hypotension,
Hypertension,
Flushing, Pulmonary embolism, Bronchospasm,
Wheezing, Cough,
Dyspnea, Vomiting, Diarrhea, Abdominal
discomfort, Nausea,
Sensation of numbness in the face, Angioedema,
Urticaria,
Pruritus, Pain at the injection site, Malaise,
Feeling hot,
Chills, Pyrexia, Chest pain, Chest discomfort,
Blood pressure
decreased.

SHIRE and the Shire Logo are registered trademarks of Shire
Pharmaceutical Holdings Ireland Limited or its affiliates.

FEIBA is a registered trademark of Baxalta Incorporated, a wholly
owned, indirect subsidiary of Shire plc.

References

1. Thompson C. et al. "Estimating the Global Annual Bleed Rate in
Haemophilia." European Association for Haemophilia and Allied
Disorders. Paris, France. February 1-3, 2017. Available at: http:/
/onlinelibrary.wiley.com/doi/10.1111/hae.13150/full#hae13150-sec-0
190
2. World Federation of Hemophilia. "About Bleeding Disorders:
Treatment." World Federation of Hemophilia website.
http://www.wfh.org/en/page.aspx?pid=642 . Accessed January 12,
2017.
3. Hartmann R. et al. "Synergistic Effects of a Procoagulant
Bispecific Antibody and FEIBA or Factor VIIA on Thrombin
Generation." European Association for Haemophilia and Allied
Disorders. Paris, France. February 1-3, 2017. Available at: http:/
/onlinelibrary.wiley.com/doi/10.1111/hae.13158/full#hae13158-sec-0
058
4. Crea R. et al. "Four Decade Cumulative Review of Thrombo-Embolic
Events Reported with the Use of Activated Prothrombin Complex
Concentrate in Congenital Haemophilia." European Association for
Haemophilia and Allied Disorders. Paris, France. February 1-3,
2017. Available at: http://onlinelibrary.wiley.com/doi/10.1111/hae
.13150/full#hae13150-sec-0012
5. World Federation of Hemophilia. "What is hemophilia?" World
Federation of Hemophilia website.
http://www.wfh.org/en/page.aspx?pid=646. Accessed January 12, 2017
6. World Federation of Hemophilia. Report on the Annual Global Survey
2015. World Federation of Hemophilia website.
http://www1.wfh.org/publication/files/pdf-1669.pdf. Accessed
January 12, 2017.
7. National Hemophilia Foundation. "Hemophilia A". National
Hemophilia Foundation website. https://www.hemophilia.org/Bleeding
-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A. Accessed
January 12, 2017.
8. World Federation of Hemophilia. "What are inhibitors?" World
Federation of Hemophilia website.
http://www.wfh.org/en/page.aspx?pid=651. Accessed January 12,
2017.

NOTES TO EDITORS

About Shire

Shire is the leading global biotechnology company focused on
serving people with rare diseases and other highly specialized
conditions. We strive to develop best-in-class products, many of
which are available in more than 100 countries, across core
therapeutic areas including Hematology, Immunology, Neuroscience,
Ophthalmics, Lysosomal Storage Disorders, Gastrointestinal / Internal
Medicine / Endocrine and Hereditary Angioedema; and a growing
franchise in Oncology.

Our employees come to work every day with a shared mission: to
develop and deliver breakthrough therapies for the hundreds of
millions of people in the world affected by rare diseases and other
high-need conditions, and who lack effective therapies to live their
lives to the fullest.

http://www.shire.com

Forward-Looking Statements

Statements included herein that are not historical facts,
including without limitation statements concerning future strategy,
plans, objectives, expectations and intentions, 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;
- increased pricing pressures and limits on patient access as a
result of governmental regulations and market developments may
affect Shire's future revenues, financial condition, and results of
operations;
- Shire conducts its own manufacturing operations for certain of its
products and is reliant on third party contract manufacturers to
manufacture other products and to provide goods and services. Some
of Shire's products or ingredients are only available from a single
approved source for manufacture. Any disruption to the supply chain
for any of Shire's products may result in Shire being unable to
continue marketing or developing a product or may result in Shire
being unable to do so on a commercially viable basis for some
period of time;
- the manufacture of Shire's products is subject to extensive
oversight by various regulatory agencies. Regulatory approvals or
interventions associated with changes to manufacturing sites,
ingredients or manufacturing processes could lead to significant
delays, an increase in operating costs, lost product sales, an
interruption of research activities or the delay of new product
launches;
- certain of Shire's therapies involve lengthy and complex processes,
which 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;
- the actions of certain customers could affect Shire's ability to
sell or market products profitably. Fluctuations in buying or
distribution patterns by such customers can adversely affect
Shire's revenues, financial conditions, or results of operations;
- Shire's products and product candidates face substantial
competition in the product markets in which it operates, including
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 combined company's revenues,
financial condition, or results of operations;
- inability to successfully compete for highly qualified personnel
from other companies and organizations;
- failure to achieve the strategic objectives with respect to Shire's
acquisition of NPS Pharmaceuticals, Inc., Dyax Corp. ("Dyax") or
Baxalta Inc. ("Baxalta") may adversely affect 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, as well as changes in
foreign currency exchange rates and interest rates, that adversely
impact the availability and cost of credit and customer purchasing
and payment patterns, including the collectability of customer
accounts receivable;
- failure of a marketed product to work effectively or if such a
product is the cause of adverse side effects could result in damage
to the Shire's reputation, the withdrawal of the product and legal
action against Shire;
- investigations or enforcement action by regulatory authorities or
law enforcement agencies relating to Shire's activities in the
highly regulated markets in which it operates may result in
significant legal costs and the payment of substantial compensation
or fines;
- 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 incurred substantial additional indebtedness to finance the
Baxalta acquisition, which may decrease its business flexibility
and increase borrowing costs;
- difficulties in integrating Dyax or Baxalta into Shire may lead to
the combined company not being able to realize the expected
operating efficiencies, cost savings, revenue enhancements,
synergies or other benefits at the time anticipated or at all; and
- other risks and uncertainties detailed from time to time in Shire's
filings with the Securities and Exchange Commission, including
those risks outlined in "ITEM 1A: Risk Factors" in Shire's
Quarterly Report on Form 10-Q for the quarter ended June 30, 2016.

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.

S27841

2/17

Investor Relations
Ian Karp ikarp@shire.com +1 781 482 9018
Robert Coates rcoates@shire.com +44 1256 894874
Media
Molly Poarch molly.poarch@shire.com +1 312 965 3413

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