Shire Announces European Approval of VPRIV(R) (velaglucerase alfa) for the Treatment of Type 1 Gaucher Disease

By Shire Plc, PRNE
Wednesday, August 25, 2010

CAMBRIDGE, Massachusetts, August 26, 2010 - Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty
biopharmaceutical company, announced today that the European Commission has
granted marketing authorisation for VPRIV(R) (velaglucerase alfa), a human
cell line derived enzyme replacement therapy (ERT) for the long-term
treatment of type 1 Gaucher disease. VPRIV has been authorized as an orphan
medicine through the Centralised Procedure, making it available in 30
countries across Europe.

This approval was based on data from Shire's velaglucerase alfa clinical
development programme which represents the largest and most comprehensive
clinical data set supporting registration for an ERT for type 1 Gaucher
disease. In total, over 100 Gaucher patients at 24 sites in 10 countries
around the world participated in the clinical studies, all of which met their
primary endpoints.

"Gaucher disease is a rare and often debilitating condition," said
Professor Tim Cox from the Department of Medicine, University of Cambridge,
and founder of the largest U.K. Gaucher clinic at Addenbrooke's Hospital.
"The European approval of VPRIV is important in that we now have an
alternative, licensed therapeutic enzyme. For type 1 patients the
availability of VPRIV provides further opportunities to individualise
treatment options for this complex disorder."

Across Europe, hundreds of type 1 Gaucher patients have been receiving
velaglucerase alfa through early access programmes, developed in partnership
with national authorities, Gaucher expert physicians and patient
associations. Globally there are over 850 patients on velaglucerase alfa and
demand continues to be strong. As a result, Shire has implemented a program
to carefully monitor demand and manage requests from physicians and patients
in order to ensure long-term, uninterrupted treatment with VPRIV.

"The Marketing Authorisation for VPRIV in the EU is an important
milestone for Shire," said Sylvie Grégoire, President, Shire Human Genetic
Therapies. "Our efforts to accelerate our manufacturing, clinical and
regulatory timelines have resulted in VPRIV's approval in Europe and the US
months ahead of schedule."


Velaglucerase alfa is made using Shire's gene-activation technology, in a
human cell line. Velaglucerase alfa has the exact human amino acid sequence
as the endogenous glucocerebrosidase enzyme and also has a human
glycosylation pattern. The safety and efficacy of velaglucerase alfa was
assessed in adults and children aged 4 years and older via a phase three
program, which included Gaucher patients who switched to velaglucerase alfa
after being treated with imiglucerase, as well as naïve patients, including
an active comparison with imiglucerase. VPRIV was approved in the United
by the Food and Drug Administration on February 26, 2010.

Important Safety Information

The most serious adverse reactions in patients treated with VPRIV were
hypersensitivity reactions.

Infusion-related reactions were the most commonly observed adverse
reactions in patients treated with VPRIV in clinical studies. The most
commonly observed symptoms of infusion-related reactions were: headache,
dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia.
Generally the infusion-related reactions were mild and, in treatment-naïve
patients, onset occurred mostly during the first 6 months of treatment and
tended to occur less frequently with time. Other commonly observed adverse
reactions in >10% of patients were: abdominal pain, back pain, joint pain,
upper respiratory tract infection, and activated partial thromboplastin time
prolonged. Adverse reactions more commonly seen in paediatric patients (>10%
difference) included upper respiratory tract infection, rash, activated
partial thromboplastin time prolonged, and pyrexia. In clinical trials one
patient developed neutralizing antibodies.

VPRIV is not available in all countries and prescribing information may
differ between countries. Please consult your local prescribing information.

About Gaucher Disease

Gaucher disease is an autosomal recessive disorder caused by mutations in
the GBA gene which results in a deficiency of the lysosomal enzyme
beta-glucocerebrosidase. This enzymatic deficiency causes an accumulation of
glucocerebroside, primarily in macrophages. In this lysosomal storage
disorder (LSD), clinical features are reflective of the distribution of
Gaucher cells in the liver, spleen, bone marrow, and other organs. The
accumulation of glucocerebrosidase in the liver and spleen leads to
organomegaly. Presence of Gaucher cells in the bone marrow and spleen lead to
clinically significant anemia and thrombocytopenia.

Gaucher disease is the most prevalent of the lysosomal storage disorders
diseases. Gaucher disease has classically been categorized into 3 clinical
types. Type 1 Gaucher disease is characterized by variability in signs,
symptoms, severity, and progression. Type 1 is the most common and is
distinguished from type 2 and type 3 by the lack of early neurological

Notes to editors


Shire's strategic goal is to become the leading specialty
biopharmaceutical company that focuses on meeting the needs of the specialist
physician. Shire focuses its business on attention deficit hyperactivity
disorder (ADHD), human genetic therapies (HGT) and gastrointestinal (GI)
diseases as well as opportunities in other therapeutic areas to the extent
they arise through acquisitions. Shire's in-licensing, merger and acquisition
efforts are focused on products in specialist markets with strong
intellectual property protection and global rights. Shire believes that a
carefully selected and balanced portfolio of products with strategically
aligned and relatively small-scale sales forces will deliver strong results.

For further information on Shire, please visit the Company's website:

ACT OF 1995

Statements included herein that are not historical facts 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, the Company's results could be
materially adversely affected. The risks and uncertainties include, but are
not limited to, risks associated with: the inherent uncertainty of research,
development, approval, reimbursement, manufacturing and commercialization of
the Company's Specialty Pharmaceutical and Human Genetic Therapies products,
as well as the ability to secure and integrate new products for
commercialization and/or development; government regulation of the Company's
products; the Company's ability to manufacture its products in sufficient
quantities to meet demand; the impact of competitive therapies on the
Company's products; the Company's ability to register, maintain and enforce
patents and other intellectual property rights relating to its products; the
Company's ability to obtain and maintain government and other third-party
reimbursement for its products; and other risks and uncertainties detailed
from time to time in the Company's filings with the Securities and Exchange

    For further information please contact:

    Investor Relations
    Eric Rojas


    Jessica Mann (Rest of the World)

    Jessica Cotrone (North America, HGT)

For further information please contact: Investor Relations, Eric Rojas, +1-781-482-0999; Media: Jessica Mann (Rest of the World), +44-1256-894-280; Jessica Cotrone (North America, HGT), +1-781-482-9538

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