Navigation Links
Shire Receives Fast Track Designation for velaglucerase alfa for Gaucher Disease

LEXINGTON, Massachusetts, July 16 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Type I Gaucher disease. Shire is working with the FDA to determine subsequent steps and timing for the filing of its NDA.

Fast Track designation is an FDA approved process that facilitates the development and expedites the review of drugs to treat serious diseases and fill an unmet medical need with the goal of getting important new treatments to patients earlier. This process allows a company to file the sections of the 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 rolling basis as the additional sections are completed and submitted for review.

Shire is completing a phase III clinical program that includes three phase III controlled studies involving over 100 patients at 24 sites in 10 countries around the world.

On July 6th, Shire announced that it filed a treatment protocol for velaglucerase alfa at the request of the FDA, which if accepted would allow physicians to treat Gaucher disease patients with velaglucerase alfa on an early access basis, ahead of commercial availability in the US. Under the conditions of the treatment protocol, Shire would provide velaglucerase alfa free of charge initially, in order to provide access to patients as quickly as possible.

Velaglucerase alfa is made with Shire's proprietary technology, in a human cell line. The enzyme produced has the exact human amino acid sequence and carries a human glycosylation pattern.

Background on Gaucher disease

Gaucher disease is an autosomal recessive disease and the most prevalent Lysosomal Storage Disorder (LSD), with an incidence of about 1 in 20,000 live births. Despite the fact that Gaucher Disease consists of a phenotype, with varying degrees of severity, it has been sub-divided in three subtypes according to the presence or absence of neurological involvement. It is also the 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 involves many organ systems, such as liver, spleen, lungs, brain, metabolism and bone marrow.

Gaucher Disease results from a specific enzyme deficiency in the body, caused by a genetic mutation received from both parents. The disease course is quite variable, ranging from no outward symptoms to severe disability and death. Carrier status can be detected through blood or saliva to identify potential carriers of the Gaucher gene. Gaucher Disease can be diagnosed early through a blood test.

Worldwide the diagnosed population of Gaucher Disease patients is approximately 7,000. Based on incidence, the estimated total world population is likely to be between 10,000 and 15,000 patients.

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:


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.

    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

    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
Copyright©2009 PR Newswire.
All rights reserved

Related medicine technology :

1. Shire has Filed a Treatment Protocol for Velaglucerase Alfa for Gaucher Disease
2. Shire Presented Results of a New Database Analysis on Lialda(R) (Mesalamine) and Other 5-ASAs for Ulcerative Colitis at Digestive Disease Week
3. Shire Announces Study Results of the Effects of INTUNIV(TM) (Guanfacine) Extended Release on Secondary Measures in Children with ADHD and Oppositional Symptoms
4. Shire Withdraws European Application for DAYTRANA(TM) (Methylphenidate Transdermal System)
5. Shire to add New Orphan Drug to its HGT Portfolio - EU Launch Imminent
6. Shire Announces Approval of ELAPRASE(R) (idursulfase) in Brazil
7. Shires Investigational Nonstimulant ADHD Treatment INTUNIV(TM) (Guanfacine Extended Release) Demonstrated Significant Efficacy in Reducing ADHD Symptoms at All Measured Time Points Up to 24 Hours Postdose
8. Shire to Present Additional Scientific Data on ADHD Treatment Portfolio at American Psychiatric Association Annual Meeting
9. Shire Enhances its Orphan Drug Pipeline With the Acquisition of a New Clinical Candidate for Metachromatic Leukodystrophy
10. Shire Strives to Increase Adherence Among Chronic Kidney Disease Stage 5 Patients
11. USC Keck School of Medicines Dr. Alan Epstein Receives Prestigious National Cancer Institute RAID Award for Breakthrough IL-2 Molecule Therapeutic
Post Your Comments:
(Date:11/30/2015)... Nov. 30, 2015  PTS Diagnostics, the U.S.-based manufacturer ... analyzers, A1CNow ® systems, and PTS Detect™ ... of patents that will propel the company into the ... Europe . The technology is a ... those on smartphones and tablets, and uses test strip ...
(Date:11/30/2015)... , Nov. 30, 2015 Cumberland Pharmaceuticals (CPIX), ... present live at on December 3, 2015. ... TIME: 3:15p.m. ET LINK: ... LINK: --> ... event where investors are invited to ask the company questions ...
(Date:11/30/2015)... LAKE CITY , Nov. 30, 2015 Booth ... (NYSE: VAR ) will exhibit a broader array of ... of the Radiological Society of North America ... Varian exhibit at the meeting will feature X-ray components "At ... CT tube, a line of products from Varian,s Claymount brand, ...
Breaking Medicine Technology:
(Date:11/30/2015)... , ... November 30, 2015 , ... ... at a University of Delaware Accounting and Management of Information Systems course. Based ... for mid-market businesses. Sommer will speak at before student in the Enterprise Resource ...
(Date:11/30/2015)... , ... November 30, 2015 , ... ... continuing education course in Dallas, TX, on January 29 and 30, 2016. The ... improve the functions of their practices, to learn how to better succeed in ...
(Date:11/30/2015)... PA (PRWEB) , ... November 30, 2015 , ... A ... 15th annual Regional Biotech Conference, organized by the Baruch S. Blumberg Institute. , The ... 100 of the area’s life science and biotechnology leaders for the conference, which focused ...
(Date:11/30/2015)... ... November 30, 2015 , ... HemoTreat™ has announced ... its hemorrhoid ointment to its website. , “Our goal is simple:” says Michael ... hemorrhoids. Adding the comparison chart and ingredient list allows our customers to quickly ...
(Date:11/30/2015)... ... November 30, 2015 , ... ChiliPad , Chili ... maximize recovery through quality sleep. Tim DiFrancesco, training coach for the LA Lakers, ... sleep. ChiliPad precisely regulates the surface temperature of each side of the bed ...
Breaking Medicine News(10 mins):