Shire Receives 2010 Corporate Award From the National Organization for Rare Disorders (NORD) for the Development of VPRIV (velaglucerase alfa for Injection)
By Shire Plc, PRNETuesday, May 18, 2010
Organization Recognizes Shire's Commitment to the Gaucher Disease Community
CAMBRIDGE, Massachusetts, May 19, 2010 - Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty
biopharmaceutical company, today announced that it has received the 2010
Partners in Progress Corporate Award from NORD. Shire was recognized for its
efforts to accelerate the development of VPRIV(TM), a human cell line derived
enzyme replacement therapy (ERT) for the treatment of Type 1 Gaucher disease,
and provide the therapy to patients ahead of commercial approval through
early access programs. The award was presented yesterday, May 18, 2010, at
NORD's Annual Gala in Washington, D.C.
This award marks the second time in the last three years that Shire has
been recognized by NORD for its contributions and ongoing commitment to
improving the lives of those affected by rare diseases. In 2007, the company
received a Corporate Leadership Award for the development of an ERT for
Hunter syndrome.
"Gaucher disease is a debilitating condition and the past several months
have been very challenging for patients and their families," said Peter L.
Saltonstall, President and CEO, NORD. "Shire provided a very important
treatment option at a time when it was greatly needed. We applaud their
responsiveness during this difficult situation."
Each year NORD honors organizations that have made a positive
contribution to further the needs of the patient community, and have inspired
the public to do so as well. Shire received the NORD Corporate Award for
VPRIV because of its work to make the drug available to patients impacted by
the ongoing disruption in supply of the only other marketed ERT for Type 1
Gaucher disease.
VPRIV was approved by the Food and Drug Administration (FDA) on February
26, 2010, as a long-term treatment for adult and pediatric patients with Type
1 Gaucher disease. It has also been granted accelerated assessment by the
European Medicines Agency (EMA) in the European Union (EU). Shire expects to
launch VPRIV in the EU in the second half of 2010, and in other countries
beginning in 2011.
"It is a tremendous honor to be recognized by NORD," said Sylvie
Gregoire, President of Shire HGT. "This award embodies the spirit of our
organization-every employee at Shire is dedicated to developing and bringing
forward new products, services and support offerings which can make a
positive impact on patients' daily lives. We look forward to collaborating
with NORD on many other important initiatives in the future."
Important Safety Information for VPRIV
The most serious adverse reactions seen 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, low or high blood pressure, nausea, tiredness and weakness, and
fever. Generally the infusion-related reactions were mild and, in
treatment-naive patients, onset occurred mostly during the first 6 months of
treatment and tended to occur less frequently with time. Adverse reactions
more commonly seen in pediatric patients compared to those observed in adult
patients (>10% difference) include rash, upper respiratory tract infection,
prolonged activated partial thromboplastin time, and fever.
As with all therapeutic proteins, there is a potential for
immunogenicity. In the clinical studies 1 of 54 treatment-naive patients
treated with VPRIV developed IgG class antibodies. It is unknown if the
presence of IgG antibodies to VPRIV is associated with a higher risk of
infusion reactions.
Full prescribing information for VPRIV can be found at
www.VPRIV.com.
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
symptoms.
Notes to editors
SHIRE PLC
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:
www.shire.com.
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For further information please contact: Investor Relations, Clea Rosenfeld (Rest of the World), +44-1256 894-160; Eric Rojas (North America), +1-781-482-0999; Media: Jessica Mann (Rest of the World), +44-1256 894-280; Jessica Cotrone (North America, HGT), +1-617-613-4640
Tags: Cambridge, Massachusetts, May 19, Shire Plc, United States of America