FDA Grants Priority Review for Shire's velaglucerase alfa for Type 1 Gaucher Disease
November 04, 2009 Shire Plc United States of America
CAMBRIDGE, Massachusetts, November 4 /PRNewswire-FirstCall/ -- Shire plc
(LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company,
today announced that the United States Food and Drug Administration (FDA) has
granted Priority Review for the New Drug Application (NDA) for velaglucerase
alfa, the company's enzyme replacement therapy in development for the
treatment of Type 1 Gaucher disease.
Priority Review designation is given to drugs that offer major advances
in treatment, or provide a treatment where no adequate therapy exists, and
accelerates the target review timing from ten to six months. The FDA has
issued an action date for the NDA of February 28, 2010 under the Prescription
Drug User Fee Act (PDUFA).
In the U.S., patients continue to be enrolled in an FDA-approved
treatment protocol, under which Gaucher patients receive velaglucerase alfa
prior to commercialization. Shire has also engaged with national and regional
authorities outside the U.S. and patients are receiving velaglucerase alfa
through pre-approval access programs. Shire confirms it is on track with its
filing 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 in
the GBA gene which results in a deficiency of the lysosomal enzyme
beta-glucocerebrosidase. This enzymatic deficiency causes an accumulation of
glucocerebroside, primarily in macrophages. In this lysosomal storage
disorder (LSD), clinical features are reflective of the distribution of
Gaucher cells in the liver, spleen, bone marrow, skeleton, and lungs. The
accumulation of glucocerebrosidase in the liver and spleen leads to
organomegaly. Bone involvement results in skeletal abnormalities and
deformities as well as bone pain crises. Deposits in the bone marrow and
splenic sequestration lead to clinically significant anemia and
thrombocytopenia.
Gaucher disease is the most prevalent lysosomal storage disorder, with an
incidence of about 1 in 20,000 live births. Gaucher disease has classically
been categorized into 3 clinical types. Type 1 is the most common; it is
distinguished from Type 2 and Type 3 by the lack of central nervous system
involvement. Type 1 Gaucher disease is characterized by variability in signs,
symptoms, severity, and progression.
Velaglucerase alfa supplements or replaces beta-glucocerebrosidase, the
enzyme that catalyzes the hydrolysis of glucocerebroside, reducing the amount
of accumulated glucocerebroside and correcting the pathophysiology of Gaucher
disease.
Shire's velaglucerase alfa program included the largest and most
comprehensive set of Phase III clinical trials conducted to date for Gaucher
disease. Over 100 patients at 24 sites in 10 countries around the world have
participated the clinical studies. Velaglucerase alfa is made using Shire's
proprietary technology, in a human cell line. The enzyme produced has the
exact human amino acid sequence and has a human glycosylation pattern.
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 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
