FDA Grants Priority Review for Shire's velaglucerase alfa for Type 1Gaucher Disease

Wed, 11/04/2009 - 3:35am
Shire plc, a global specialty biopharmaceutical company,today announced that the FDA hasgranted Priority Review for the New Drug Application (NDA) for velaglucerasealfa, the company's enzyme replacement therapy in development for thetreatment of Type 1 Gaucher disease. Priority Review designation is given to drugs that offer major advancesin treatment, or provide a treatment where no adequate therapy exists, andaccelerates the target review timing from ten to six months. The FDA hasissued an action date for the NDA of February 28, 2010 under the PrescriptionDrug User Fee Act (PDUFA). In the U.S., patients continue to be enrolled in an FDA-approvedtreatment protocol, under which Gaucher patients receive velaglucerase alfaprior to commercialization. Shire has also engaged with national and regionalauthorities outside the U.S. and patients are receiving velaglucerase alfathrough pre-approval access programs. Shire confirms it is on track with itsfiling of the Marketing Authorization Application (MAA) in the EU for 2009. Gaucher disease is an autosomal recessive disorder caused by mutations inthe GBA gene which results in a deficiency of the lysosomal enzymebeta-glucocerebrosidase. This enzymatic deficiency causes an accumulation ofglucocerebroside, primarily in macrophages. In this lysosomal storagedisorder (LSD), clinical features are reflective of the distribution ofGaucher cells in the liver, spleen, bone marrow, skeleton, and lungs. Theaccumulation of glucocerebrosidase in the liver and spleen leads toorganomegaly. Bone involvement results in skeletal abnormalities anddeformities as well as bone pain crises. Deposits in the bone marrow andsplenic sequestration lead to clinically significant anemia andthrombocytopenia. Gaucher disease is the most prevalent lysosomal storage disorder, with anincidence of about 1 in 20,000 live births. Gaucher disease has classicallybeen categorized into 3 clinical types. Type 1 is the most common; it isdistinguished from Type 2 and Type 3 by the lack of central nervous systeminvolvement. Type 1 Gaucher disease is characterized by variability in signs,symptoms, severity, and progression. Velaglucerase alfa supplements or replaces beta-glucocerebrosidase, theenzyme that catalyzes the hydrolysis of glucocerebroside, reducing the amountof accumulated glucocerebroside and correcting the pathophysiology of Gaucherdisease. Shire's velaglucerase alfa program included the largest and mostcomprehensive set of Phase III clinical trials conducted to date for Gaucherdisease. Over 100 patients at 24 sites in 10 countries around the world haveparticipated the clinical studies. Velaglucerase alfa is made using Shire'sproprietary technology, in a human cell line. The enzyme produced has theexact human amino acid sequence and has a human glycosylation pattern.

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