London, Nov. 4, 2009 (PR Newswire UK Disclose) --
FDA Grants Priority Review for Shire's velaglucerase alfa for Type 1 Gaucher
Disease
FDA issued action date of February 28, 2010 under the Prescription Drug User
Free Act (PDUFA)
Cambridge, MA - November 4, 2009 - 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.
