Navigation Links
Shire has Filed a Treatment Protocol for Velaglucerase Alfa for Gaucher Disease
Date:7/6/2009

LEXINGTON, Massachusetts, July 6 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces that, at the request of the FDA, in view of a potential restriction on the availability of the current approved and marketed treatment for Gaucher Disease patients, it has filed a treatment protocol for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Gaucher Disease.

If approved by the FDA, the treatment protocol would allow physicians to treat Gaucher Disease patients with velaglucerase alfa 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.

Shire has sufficient supply of velaglucerase alfa to meet anticipated patient demand.

Shire is working with the FDA to file a New Drug Application (NDA) for velaglucerase alfa as early as possible.

Shire will provide further updates when available via press releases and/or its website (http://www.shire.com) on its progress with its discussions with the FDA and other regulatory bodies about the development of velaglucerase alfa as an alternative choice for Gaucher Disease patients.

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.

Notes to editors

SHIRE PLC

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: http://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.

    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
Copyright©2009 PR Newswire.
All rights reserved

Related medicine technology :

1. Shire Presented Results of a New Database Analysis on Lialda(R) (Mesalamine) and Other 5-ASAs for Ulcerative Colitis at Digestive Disease Week
2. Shire Announces Study Results of the Effects of INTUNIV(TM) (Guanfacine) Extended Release on Secondary Measures in Children with ADHD and Oppositional Symptoms
3. Shire Withdraws European Application for DAYTRANA(TM) (Methylphenidate Transdermal System)
4. Shire to add New Orphan Drug to its HGT Portfolio - EU Launch Imminent
5. Shire Announces Approval of ELAPRASE(R) (idursulfase) in Brazil
6. 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
7. Shire to Present Additional Scientific Data on ADHD Treatment Portfolio at American Psychiatric Association Annual Meeting
8. Shire Enhances its Orphan Drug Pipeline With the Acquisition of a New Clinical Candidate for Metachromatic Leukodystrophy
9. Shire Strives to Increase Adherence Among Chronic Kidney Disease Stage 5 Patients
10. Shareholder Class Action Filed Against Rigel Pharmaceuticals, Inc. by the Law Firm of Barroway Topaz Kessler Meltzer & Check, LLP
11. Shareholder Class Action Filed Against Vertex Pharmaceuticals Incorporated by the Law Firm of Schiffrin Barroway Topaz & Kessler, LLP
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/23/2016)... , June 23, 2016 Any dentist who ... challenges of the current process. Many of them do not ... the technical difficulties and high laboratory costs involved. And those ... offer it at such a high cost that the majority ... Dr. Parsa Zadeh , founder of Dental ...
(Date:6/23/2016)... , June 23, 2016 Roche (SIX: RO, ... clearance for its Elecsys BRAHMS PCT (procalcitonin) assay as ... or septic shock. With this clearance, Roche is the ... fully integrated solution for sepsis risk assessment and management. ... bacterial infection and PCT levels in blood can aid ...
(Date:6/23/2016)... -- Research and Markets has announced the addition ... to their offering. The ... commercial environment for MedImmune to enter. The US ageing population ... to drive considerable growth for effective anti-influenza medications. The introduction ... considerably, but development is still in its infancy. ...
Breaking Medicine Technology:
(Date:6/27/2016)... California (PRWEB) , ... June 27, 2016 , ... "FCPX ... fully customizable inside of Final Cut Pro X," said Christina Austin - CEO of ... unique style. Final Cut Pro X users can now reveal the media ...
(Date:6/26/2016)... ... 2016 , ... Quality metrics are proliferating in cancer care, and are derived ... eye of the beholder, according to experts who offered insights and commentary in the ... Care. For the full issue, click here . , For the American Society ...
(Date:6/26/2016)... ... June 26, 2016 , ... ... have been diagnosed with endometriosis. These women need a treatment plan to not ... comprehensive approach that can help for preservation of fertility and ultimately achieving a ...
(Date:6/25/2016)... ... June 25, 2016 , ... First Choice Emergency ... named Dr. Sesan Ogunleye, as the Medical Director of its new Mesquite-Samuell Farm facility. ... Director of our new Mesquite location,” said Dr. James M. Muzzarelli, Executive Medical Director ...
(Date:6/24/2016)... Los Angeles, CA (PRWEB) , ... June 24, 2016 , ... ... plastic surgery procedures that most people are unfamiliar with. The article goes on to ... known procedures, but also many of these less common operations such as calf and ...
Breaking Medicine News(10 mins):