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Shire To Present Important Study Findings For Gaucher Disease Treatment And GLD Development Program At The Lysosomal Disease Network (LDN) World Symposium

February 3, 2010

Data sponsored by the Canadian Fabry Disease Initiative (CFDI) to be presented by Dr. Michael West, Dalhousie University

Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today announced that data from one of three Phase III clinical trials for velaglucerase alfa, the company’s enzyme replacement therapy (ERT) in development for the treatment of Type 1 Gaucher disease, will be presented at the Lysosomal Disease Network (LDN) World Symposium, February 10-12, 2010 in Miami, Florida. In addition to two oral presentations and two poster presentations on Gaucher disease, the company will present findings from a re-analysis of disease stage progression in Globoid Cell Leukodystrophy (GLD). Dr. Michael West from Dalhousie University in Halifax, Canada will also present head-to-head REPLAGAL ® 0.2 mg/kg and Fabrazyme 1.0 mg data, sponsored by the Canadian Fabry Disease Initiative (CFDI).

“We are excited to participate in this very important scientific meeting,” said Whaijen Soo, MD, PhD, Senior Vice President, Research and Development, Shire Human Genetic Therapies (HGT). “Shire continues to deliver on its promise to pursue the development and commercialization of treatments which will enable people with life-altering conditions to lead better lives – and we’re looking forward to sharing data which underscores the success of our commitment in the area of rare genetic diseases.”

A summary of the key scientific presentations is provided below. Information about these data presentations mentioned in this release is embargoed until the respective presentation sessions have taken place at the meeting.

Gaucher disease

  • February 10, 2010; 4-6 PM EST
    Enzyme Replacement Therapy with velaglucerase alfa Improves Key Clinical Parameters in a Pediatric Subgroup with Type 1 Gaucher Disease
  • February 10, 2010; 4-6 PM EST
    Five-Year Safety and Efficacy of velaglucerase alfa in Gaucher Disease Type 1: Experience in Clinic and Home Settings
  • February 11, 2010; 9:30 AM EST
    Enzyme Replacement Therapy with velaglucerase alfa Significantly Improves Key Clinical Parameters in Type 1 Gaucher disease: Positive Results from a Randomized, Double-blind, Global Phase III Study (Oral Presentation)
  • February 11, 2010; 9:45 AM EST
    Antigenic Differences in Patients Receiving Velaglucerase alfa or Imiglucerase (Oral Presentation)

Globoid Cell Leukodystrophy

  • February 11, 2010; 3:30 PM EST
    A Re-analysis of Disease Stage Progression in Krabbe Disease (infantile Globoid Cell Leukodystrophy, iGLD) (Oral Presentation)

Fabry disease

  • February 11, 2010; 11:00 AM EST
    A Randomized Controlled Trial of Enzyme Replacement Therapy in Fabry Disease: The Canadian Fabry Disease Initiative at Year Three (Oral Presentation)


About REPLAGAL® (agalsidase alfa)
REPLAGAL is a human form of enzyme alpha-galactosidase A (a-Gal A) manufactured in a human cell line by gene activation.  REPLAGAL is the only human-cell-line-derived form of enzyme replacement therapy (ERT) that is indicated for the long-term treatment of patients with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency).

REPLAGAL first received marketing authorization in the European Union in 2001, and is approved in 45 countries, but not the U.S. On December 22, 2009 Shire submitted a Biologics License Application with the FDA for REPLAGAL.

About velaglucerase alfa
Velaglucerase alfa is an investigational enzyme replacement therapy for Type 1 Gaucher disease. Velaglucerase alfa is made using Shire’s gene activation technology, in a human cell line. The enzyme produced has the exact human amino acid sequence and has a human glycosylation pattern.

The FDA has issued an action date for the NDA of velaglucerase alfa of February 28, 2010 under the Prescription Drug User Fee Act (PDUFA).

About Fabry disease
Fabry disease is a lysosomal storage disorder (LSD) that interferes with the body’s ability to break down a specific fatty substance (globotriaosylceramide or Gb3) which accumulates within the body due to deficiency of a specific enzyme (α-galactosidase A).

Fabry disease affects both males and females and can present with a number of signs or symptoms of variable degree, such as cardiovascular and/or renal dysfunction, intense or burning pain, heat intolerance, skin lesions, gastrointestinal complaints, hearing loss, and ocular problems.

Lifespan is typically reduced in patients with Fabry disease by approximately 20 years in men and 15 years in women, compared with the general population. The principal causes of death are renal failure, cardiomyopathy and cerebrovascular events (e.g. stroke).

Fabry disease affects an estimated 8,000 to 10,000 people worldwide.

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, skeleton, and lungs. The accumulation of glucocerebrosidase in the liver and spleen leads to organomegaly. Bone involvement results in skeletal abnormalities and deformities as well as bone pain crises. Deposits in the bone marrow and splenic sequestration lead to clinically significant anemia and thrombocytopenia.

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

About Globoid Cell Leukodystrophy (GLD)
Globoid cell leukodystrophy (GLD) is a rare autosomal recessive and rapidly progressive neurological disorder involving the white matter of the peripheral and central nervous systems. GLD is caused by deficiency of galactosylceramidase (GALC) activity. GALC is responsible for the lysosomal hydrolysis of certain galactolipids located mainly in myelin, galactosylceramide and psychosine (galactosylsphingosine). The pathological changes observed in GLD, including the presence of globoid cells (macrophages containing undigested galactosylceramide) in white matter, loss of oligodendrocytes and decreased myelin, appear to result from the toxic nature of psychosine and accumulation of galactosylceramide that cannot be degraded due to the GALC deficiency.    Infantile onset of GLD is often referred to as Krabbe disease.


For further information please contact:


Jessica Mann (Rest of the World)

+44 1256 894 280


Jessica Cotrone (North America, HGT)

+1 781 482 9538

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.



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.