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Priority Review designation is given to drugs that offer major advancesin treatment, or provide a treatment where no adequate therapy exists, andaccelerates the target review timing from ten to six months. The FDA hasissued an action date for the NDA of February 28, 2010 under the PrescriptionDrug User Fee Act (PDUFA).
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In the U.S., patients continue to be enrolled in an FDA-approvedtreatment protocol, under which Gaucher patients receive velaglucerase alfaprior to commercialization. Shire has also engaged with national and regionalauthorities outside the U.S. and patients are receiving velaglucerase alfathrough pre-approval access programs. Shire confirms it is on track with itsfiling of the Marketing Authorization Application (MAA) in the EU for 2009.
Background on Gaucher disease
Gaucher disease is an autosomal recessive disorder caused by mutations inthe GBA gene which results in a deficiency of the lysosomal enzymebeta-glucocerebrosidase. This enzymatic deficiency causes an accumulation ofglucocerebroside, primarily in macrophages. In this lysosomal storagedisorder (LSD), clinical features are reflective of the distribution ofGaucher cells in the liver, spleen, bone marrow, skeleton, and lungs. Theaccumulation of glucocerebrosidase in the liver and spleen leads toorganomegaly. Bone involvement results in skeletal abnormalities anddeformities as well as bone pain crises. Deposits in the bone marrow andsplenic sequestration lead to clinically significant anemia andthrombocytopenia.
Gaucher disease is the most prevalent lysosomal storage disorder, with anincidence of about 1 in 20,000 live births. Gaucher disease has classicallybeen categorized into 3 clinical types. Type 1 is the most common; it isdistinguished from Type 2 and Type 3 by the lack of central nervous systeminvolvement. Type 1 Gaucher disease is characterized by variability in signs,symptoms, severity, and progression.
Velaglucerase alfa supplements or replaces beta-glucocerebrosidase, theenzyme that catalyzes the hydrolysis of glucocerebroside, reducing the amountof accumulated glucocerebroside and correcting the pathophysiology of Gaucherdisease.
Shire's velaglucerase alfa program included the largest and mostcomprehensive set of Phase III clinical trials conducted to date for Gaucherdisease. Over 100 patients at 24 sites in 10 countries around the world haveparticipated the clinical studies. Velaglucerase alfa is made using Shire'sproprietary technology, in a human cell line. The enzyme produced has theexact human amino acid sequence and has a human glycosylation pattern.
Notes to editors
SHIRE PLC
Shire's strategic goal is to become the leading specialtybiopharmaceutical company that focuses on meeting the needs of the specialistphysician. Shire focuses its business on attention deficit hyperactivitydisorder (ADHD), human genetic therapies (HGT) and gastrointestinal (GI)diseases as well as opportunities in other therapeutic areas to the extentthey arise through acquisitions. Shire's in-licensing, merger and acquisitionefforts are focused on products in specialist markets with strongintellectual property protection and global rights. Shire believes that acarefully selected and balanced portfolio of products with strategicallyaligned and relatively small-scale sales forces will deliver strong results.
For further information on Shire, please visit the Company's website:http://www.shire.com.
"SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORMACT OF 1995
Statements included herein that are not historical facts areforward-looking statements. Such forward-looking statements involve a numberof risks and uncertainties and are subject to change at any time. In theevent such risks or uncertainties materialize, the Company's results could bematerially adversely affected. The risks and uncertainties include, but arenot limited to, risks associated with: the inherent uncertainty of research,development, approval, reimbursement, manufacturing and commercialization ofthe Company's Specialty Pharmaceutical and Human Genetic Therapies products,as well as the ability to secure and integrate new products forcommercialization and/or development; government regulation of the Company'sproducts; the Company's ability to manufacture its products in sufficientquantities to meet demand; the impact of competitive therapies on theCompany's products; the Company's ability to register, maintain and enforcepatents and other intellectual property rights relating to its products; theCompany's ability to obtain and maintain government and other third-partyreimbursement for its products; and other risks and uncertainties detailedfrom time to time in the Company's filings with the Securities and ExchangeCommission.For further information please contact: Investor Clea Rosenfeld (Rest of the World) +44-1256-894-160 Relations Eric Rojas (North America) +1-617-551-9715 Media Jessica Mann (Rest of the World) +44-1256-894-280 Jessica Cotrone (North America, HGT) +1-617-613-4640
SOURCE Shire Plc
