Now available, VPRIV replaces missing enzyme in people with rare genetic disorder
Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces that Health Canada has approved VPRIV (velaglucerase alfa), an enzyme replacement therapy (ERT) for long-term use in pediatric and adult type 1 Gaucher disease. This approval was based on data from Shire’s velaglucerase alfa clinical development programme which represents the largest and most comprehensive clinical data set supporting registration for an ERT for type 1 Gaucher disease. In total, over 100 Gaucher patients at 24 sites in 10 countries around the world participated in the clinical studies, all of which met their primary endpoints.
"Gaucher disease is a complex disorder which can be very debilitating,” said Dr. Dominick Amato, Director of the Centre for Gaucher Disease, Mount Sinai Hospital, Toronto, Ontario. “The Canadian approval of VPRIV provides us with an important additional option which helps us further customize patient treatment."
ERT is a treatment approach used for certain genetic disorders, specifically lysosomal storage disorders, in which the patient is treated with the specific enzyme they are lacking. Treating type 1 Gaucher disease requires a specific enzyme that is needed to break down certain fats, which in people with the disease, is either missing, produced in only small amounts, or unable to function properly. The parts of the body most affected include the spleen, liver, and bones.
VPRIV has the same amino acid sequence as the naturally occurring human enzyme glucocerebrosidase and is generated using Shire’s proprietary gene-activation technology in a human cell line.
"Gaucher disease symptoms can vary in severity and though not curable, certain symptoms can be treated with enzyme replacement therapy. This gives an alternative choice for people in Canada living with type 1 Gaucher disease," said Christine White, President of The National Gaucher Foundation of Canada. "We are dedicated to ensuring all new treatment options are available to all Canadians living with type 1 Gaucher disease who may benefit from it."
About 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 bet 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 glucocerebroside in the liver and spleen leads to organomegaly. Presence of Gaucher cells in the bone marrow and spleen can lead to clinically significant anemia and thrombocytopenia.
Gaucher disease is the most prevalent of the lysosomal storage disorders diseases. Gaucher disease has classically been categorized into 3 clinical types. Type 1 Gaucher disease is characterized by variability in signs, symptoms, severity, and progression. Type 1 is the most common and is distinguished from type 2 and type 3 by the lack of early neurological symptoms.
VPRIV® (velaglucerase alfa) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with Type 1 Gaucher disease. The safety and efficacy of VPRIV (velaglucerase alfa) were assessed in 5 clinical studies in a total of 94 patients with type 1 Gaucher disease who were age 2 years and older. Studies 025, 032, and 039 were conducted in patients naïve to enzyme replacement therapy. Study 025EXT was an extension to Study 025. A treatment naïve patient was defined differently for each study. Study 034 was conducted in patients who were receiving imiglucerase treatment. In these studies, VPRIV was administered intravenously over 60 minutes at doses ranging from 15 Units/kg to 60 Units/kg every other week.
About Shire plc
Shire plc’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 and 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. The structure is sufficiently flexible to allow Shire to target new therapeutic areas to the extent opportunities arise through acquisitions. Shire’s in-licensing, merger and acquisition efforts are focused on products in niche 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 about Shire, please visit the Company’s website: www.shirecanada.com
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