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 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 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
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
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