Shire Announces EMA Validation of Veyvondi[TM] [Von Willebrand Factor (Recombinant)] Marketing Authorization Application for Treatment of Von Willebrand Disease

Zug, Switzerland (ots/PRNewswire) -

VEYVONDI is the first and only recombinant von Willebrand factor
treatment for adults with von Willebrand disease, the most common
inherited bleeding disorder[1],[2],[3]

Shire plc (LSE: SHP, NASDAQ: SHPG), the leading biotechnology
company focused on serving individuals with rare diseases, today
announced that the European Medicines Agency (EMA) validated the
Marketing Authorization Application (MAA) for VEYVONDI to prevent and
treat bleeding episodes and peri-operative bleeding in adults (age 18
and older) diagnosed with von Willebrand Disease (VWD), the most
common inherited bleeding disorder. Currently available in the U.S.
as VONVENDI® [von Willebrand factor (Recombinant)], VEYVONDI is the
first and only recombinant von Willebrand factor (rVWF) treatment for
adults living with VWD.[1],[2],[3]

"With its clinical profile and physiology, VEYVONDI is a
first-of-its-kind treatment for VWD," said Alice Dietrich, MD, Head
of Global Medical Affairs, Shire. "We're deeply committed to
advancing standards of care and outcomes for VWD patients worldwide,
and today's submission marks an important milestone in our efforts to
address needs of patients living with VWD and make VEYVONDI available
to more patients who could benefit from its use."

Patients with VWD have a deficiency or dysfunction of VWF, a blood
protein required for proper clotting.[4] Because of this, the blood
does not clot properly, which may result in heavy menstrual periods,
easy bruising, or frequent nose bleeds.[4] VEYVONDI is an innovative
VWF replacement therapy produced and formulated without the addition
of any exogenous raw materials of human or animal origin, resulting
in a product that contains only trace amounts of FVIII.[2] This
provides physicians with the flexibility to manage VWF levels.[3]

VEYVONDI was studied in patients, 18 to 64 years of age, in a
multi-center, open label, non-randomized study assessing safety,
efficacy and pharmacokinetics, with and without rFVIII.[2],[3] It was
also studied in patients, 18 years and older, in a prospective,
uncontrolled, international, multi-center, open label, non-randomized
study assessing control of hemostasis before, during or after
surgical procedures, with or without rFVIII.[5] The most common
adverse reaction observed was generalized pruritus (itching).[3],[6]
The EMA filing is based on data from these two Phase 3 clinical
trials shared publicly in December 2015 and December 2016,
respectively.

About von Willebrand disease (VWD)

VWD is the most common inherited bleeding disorder worldwide,
affecting up to 1 percent of the world's population.[7] VWD is caused
by a deficiency or dysfunction of VWF, resulting in impaired blood
clotting, and it affects women and men equally.[4],[8] The disease
can manifest through various bleeding events, including heavy
menstrual periods, easy bruising or frequent nose bleeds.[4] Bleeding
caused by VWD is unpredictable and varies greatly among patients with
the disease.[8] Many people with VWD may not know that they have it
because their symptoms are mild or change over time.[9] Research
shows that as many as 9 out of 10 people with VWD have not been
diagnosed.[9]

About VONVENDI in the United States

VONVENDI was approved by the U.S. Food and Drug Administration
(FDA) in December 2015 and is indicated for on-demand treatment and
control of bleeding episodes in adults (18 years and older) with
VWD.[1] VONVENDI is the first and only rVWF treatment developed for
people living with VWD.[1] Shire is also seeking prophylaxis and
pediatric indications for VONVENDI, with trials anticipated to
conclude in 2019 and 2020, respectively. For more information on
VONVENDI, please visit vonvendi.com.

VONVENDI [von Willebrand factor (Recombinant)] Important
Information

Indication

VONVENDI [von Willebrand factor (Recombinant)] is a recombinant
von Willebrand factor indicated for on-demand treatment and control
of bleeding episodes in adults (age 18 and older) diagnosed with von
Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS

VONVENDI is contraindicated in patients who have had
life-threatening hypersensitivity reactions to VONVENDI or
constituents of the product (tri-sodium citrate-dihydrate, glycine,
mannitol, trehalose-dihydrate, polysorbate 80, and hamster or mouse
proteins).

WARNINGS AND PRECAUTIONS
Embolism and Thrombosis

Thromboembolic reactions, including disseminated intravascular
coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial
infarction, and stroke, can occur, particularly in patients with
known risk factors for thrombosis. Monitor for early signs and
symptoms of thrombosis such as pain, swelling, discoloration,
dyspnea, cough, hemoptysis, and syncope.

In patients requiring frequent doses of VONVENDI with recombinant
factor VIII, monitor plasma levels for FVIII:C activity because an
excessive rise in factor VIII levels can increase the risk of
thromboembolic complications.

Hypersensitivity Reactions

Hypersensitivity reactions, including anaphylaxis, may occur.
Symptoms can include anaphylactic shock, generalized urticaria,
angioedema, chest tightness, hypotension, shock, lethargy, nausea,
vomiting, paresthesia, pruritus, restlessness, wheezing and/or acute
respiratory distress. If signs and symptoms of severe allergic
reactions occur, immediately discontinue administration of VONVENDI
and provide appropriate supportive care.

Neutralizing Antibodies

Neutralizing antibodies (inhibitors) to von Willebrand factor
and/or factor VIII can occur. If the expected plasma levels of VWF
activity (VWF:RCo) are not attained, perform an appropriate assay to
determine if anti-VWF or anti-FVIII inhibitors are present. Consider
other therapeutic options and direct the patient to a physician with
experience in the care of either von Willebrand disease or hemophilia
A.

In patients with high levels of inhibitors to VWF or factor VIII,
VONVENDI therapy may not be effective and infusion of this protein
may lead to severe hypersensitivity reactions. Since inhibitor
antibodies can occur concomitantly with anaphylactic reactions,
evaluate patients experiencing an anaphylactic reaction for the
presence of inhibitors.

ADVERSE REACTIONS

The most common adverse reaction observed in >=2% of subjects in
clinical trials (n=66) was generalized pruritus.

For Full Prescribing Information, visit
http://www.shirecontent.com/PI/PDFs/VONVENDI_USA_ENG.pdf

SHIRE and the Shire Logo are registered trademarks of Shire
Pharmaceutical Holdings Ireland Limited or its affiliates. VEYVONDI
and VONVENDI are trademarks or registered trademarks of Baxalta
Incorporated, a wholly owned, indirect subsidiary of Shire plc.

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, among other
things, 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, including expected
operating efficiencies, cost savings, revenue enhancements,
synergies or other benefits at the time anticipated or at all with
respect to Shire's acquisition of NPS Pharmaceuticals Inc., Dyax
Corp. or Baxalta Incorporated 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 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; 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 "ITEM 1A: Risk Factors",
and in 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. "FDA approved first recombinant von Willebrand factor to treat
bleeding episodes." FDA website. https://www.fda.gov/newsevents/ne
wsroom/pressannouncements/ucm476065. Accessed June 21, 2017.
2. VONVENDI Prescribing Information.
3. Gill JC, Castaman G, Windyga J, et al. Hemostatic efficacy,
safety, and pharmacokinetics of a recombinant von Willebrand
factor in severe von Willebrand disease. Blood.
2015;126(17):2038-2046.
4. Nichols WL, Hultin MB, James AH, et al. von Willebrand disease
(VWD): evidence-based diagnosis and management guidelines, the
National Heart, Lung, and Blood Institute (NHLBI) Expert Panel
report (USA). Haemophilia. 2008;14(2):171-232.
5. Baxalta. Recombinant von Willebrand factor in subjects with severe
von Willebrand disease undergoing surgery. Available from:
https://clinicaltrials.gov/ct2/show/NCT02283268. Accessed June 16,
2017.
6. Shire Data on File.
7. James PD, Goodeve AC. von Willebrand Disease. Genet Med.
2011;13(5):365-76.
8. Leebeek FW, Eikenboom JC. Von Willebrand's Disease. N Engl J Med.
2016;375(21):2067-2080.
9. What is von Willebrand disease (VWD)? World Federation of
Hemophilia website. https://www.wfh.org/en/page.aspx?pid=673.
Accessed May 3, 2017.

ots Originaltext: Shire Pharmaceuticals Group Plc
Im Internet recherchierbar: http://www.presseportal.de

Contact:
, please contact:
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
+1-312-965-3414

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