Health Canada approves VPRIV enzyme replacement therapy for type 1 Gaucher disease

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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."



  1. Tom Hennessy Tom Hennessy Canada says:

    Gaucher's disease is very common in people disposed to polycythemia. One might wonder what connection is there between increased red blood cell production / polycythemia and Gaucher's disease.
    "High Incidence of Myeloproliferative Disorders in Ashkenazi Jews"
    "Ashkenazi Jewish ancestry (may have increased frequency of polycythemia"
    "Gaucher disease is not as well known as Tay-Sachs, it can affect anyone and is the most common Lysosomal Storage Disorder among the Ashkenazi Jewish"

The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
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