Shire Submits European Marketing Authorization Application (MAA) for velaglucerase alfa for the Treatment of Type 1 Gaucher Disease
By Shire Plc, PRNEMonday, November 23, 2009
Committee for Medicinal Products for Human Use (CHMP) Grants Accelerated Assessment
DUBLIN, November 24 - Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty
biopharmaceutical company, today announced that it has submitted a MAA to the
European Medicines Agency for velaglucerase alfa, the company's enzyme
replacement therapy in development for the treatment of Type 1 Gaucher
disease. This is the third marketing application for velaglucerase alfa that
has been submitted, with previous submissions in the United States and
Canada.
Based on a global supply shortage of the currently approved and marketed
treatment for patients with Gaucher disease, and positive results from all
three velaglucerase alfa Phase III trials, CHMP has accepted the company's
request for an accelerated assessment of the velaglucerase alfa MAA. The MAA
review is expected to begin in the December cycle. Under accelerated
assessment, the review timeline of the MAA is shortened from 210 days to 150
days.
"Gaucher disease is a debilitating condition and the continuing
imiglucerase supply shortage has had a significant impact on patients who
have lacked an alternative supply of enzyme therapy," said Timothy Cox, M.D.,
Professor of Medicine at the University of Cambridge and the founder of the
National Centre for the Treatment of Gaucher disease at Addenbrooke's
Hospital. "Shire's partnership with health regulators and physicians to
devise and implement expanded access programs for velaglucerase alfa is
greatly appreciated by treating physicians and the Gaucher community at
large. We welcome the news of the submission of the velaglucerase alfa MAA in
Europe."
In Europe and other countries outside the U.S. patients continue to
receive velaglucerase alfa through pre-approval access programs that were
developed in partnership with national and regional authorities and designed
specifically to address the continuing supply shortage. In the U.S., patients
continue to be enrolled in an FDA-approved treatment protocol that has been
open since September 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 LSD. 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 early
neurological symptoms. Type 1 Gaucher disease is characterized by variability
in signs, symptoms, severity, and progression.
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 in 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:
www.shire.com.
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For further information please contact:
Investor Cléa 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
For further information please contact: Investor Relations, Cléa 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; Jessica Cotrone (North America, HGT) +1-617-613-4640
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