LEXINGTON, Massachusetts, July 16 Shire plc(LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company,announces it has received Fast Track designation from the U.S. Food and DrugAdministration (FDA) for velaglucerase alfa, its enzyme replacement therapyin development for the treatment of Type I Gaucher disease. Shire is workingwith the FDA to determine subsequent steps and timing for the filing of itsNDA.
Fast Track designation is an FDA approved process that facilitates thedevelopment and expedites the review of drugs to treat serious diseases andfill an unmet medical need with the goal of getting important new treatmentsto patients earlier. This process allows a company to file the sections ofthe NDA as they become available instead of filing all the sections at once.It also enables the agency to commence its review and proceed on a rollingbasis as the additional sections are completed and submitted for review.
Shire is completing a phase III clinical program that includes threephase III controlled studies involving over 100 patients at 24 sites in 10countries around the world.
On July 6th, Shire announced that it filed a treatment protocol forvelaglucerase alfa at the request of the FDA, which if accepted would allowphysicians to treat Gaucher disease patients with velaglucerase alfa on anearly access basis, ahead of commercial availability in the US. Under theconditions of the treatment protocol, Shire would provide velaglucerase alfafree of charge initially, in order to provide access to patients as quicklyas possible.
Velaglucerase alfa is made with Shire's proprietary technology, in ahuman cell line. The enzyme produced has the exact human amino acid sequenceand carries a human glycosylation pattern.
Background on Gaucher disease
Gaucher disease is an autosomal recessive disease and the most prevalentLysosomal Storage Disorder (LSD), with an incidence of about 1 in 20,000 livebirths. Despite the fact that Gaucher Disease consists of a phenotype, withvarying degrees of severity, it has been sub-divided in three subtypesaccording to the presence or absence of neurological involvement. It is alsothe most common genetic disease affecting Ashkenazi Jewish people (Eastern,Central and Northern European ancestry), with a carrier frequency of 1 in 10(Dr. John Barranger and Dr. Ed Ginns 1989). This panethnic disease involvesmany organ systems, such as liver, spleen, lungs, brain, metabolism and bonemarrow.
Gaucher Disease results from a specific enzyme deficiency in the body,caused by a genetic mutation received from both parents. The disease courseis quite variable, ranging from no outward symptoms to severe disability anddeath. Carrier status can be detected through blood or saliva to identifypotential carriers of the Gaucher gene. Gaucher Disease can be diagnosedearly through a blood test.
Worldwide the diagnosed population of Gaucher Disease patients isapproximately 7,000. Based on incidence, the estimated total world populationis likely to be between 10,000 and 15,000 patients.
Notes to Editors
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 Relations Clea Rosenfeld (Rest of the World), +44-1256-894-160 Eric Rojas (North America), +1-617-551-9715 Media Jessica Mann (Rest of the World), +44-1256-894-280 Matthew Cabrey (North America), +1-484-595-8248 Jessica Cotrone (North America), +1-617-613-4640
SOURCE Shire Plc