19 May 2010
Shire Receives 2010 Corporate Award from the National Organization for Rare Disorders (NORD) for the Development of VPRIV (velaglucerase alfa for injection)
Cambridge, MA, US – 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™, 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 Grégoire, 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-naïve 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-naïve 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.
For further information please contact:
Cléa Rosenfeld (Rest of the World) +44 1256 894 160
Eric Rojas (North America) +1 781 482 0999
Jessica Mann (Rest of the World) +44 1256 894 280
Jessica Cotrone (North America, HGT) +1 617 613 4640
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: www.shire.com.
"SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM 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 Commission.