FDA Approval of VPRIV™ (Velaglucerase alfa for injection) for the Treatment of Type 1 Gaucher Disease
February 26, 2010
Shire Human Genetics, the global specialty biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for VPRIV, a human cell line derived enzyme replacement therapy (ERT) for the long-term treatment of Type 1 Gaucher disease in pediatric and adult patients. The FDA designated VPRIV for Priority Review and granted marketing approval in just 6 months. VPRIV offers patients and their physicians a new treatment option at a critical time, as the supply of the previously approved ERT for Gaucher disease is uncertain and remains disrupted.
VPRIV™ (velaglucerase alfa for injection) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy for pediatric and adult patients with Type 1 Gaucher disease “The last 6 months have been very challenging for the entire Gaucher community, and the approval of VPRIV brings an important new treatment option to patients suffering from Type 1 Gaucher disease,” said Rhonda Buyers, CEO / Executive Director, National Gaucher Foundation (NGF). “We at the NGF are excited about this approval, and by the steps that Shire has taken to improve access to treatments for patients with life-altering conditions. More about VPRIV
The VPRIV application has also been granted accelerated assessment by the European Medicines Agency (EMA) in the European Union (EU). Shire expects to launch VPRIV in the EU by the end of 2010 and in other countries beginning in 2011. Clinical Trial Results
Shire’s VPRIV clinical trial program included the largest and most comprehensive set of Phase III trials conducted to date for Gaucher disease. The efficacy of VPRIV was assessed in three clinical studies in a total of 99 patients with type 1 Gaucher disease. Eighty-two patients age 4 years and older received VPRIV and 17 patients age 3 years and older received imiglucerase. Studies I and II were conducted in patients who were not currently receiving Gaucher disease-specific therapy. Study III was conducted in patients who were receiving imiglucerase treatment immediately before starting VPRIV. In these studies, VPRIV was administered intravenously over 60 minutes at doses ranging from 15 Units/kg to 60 Units/kg every other week. Each study met its primary endpoint. 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 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, and other organs. The accumulation of glucocerebrosidase in the liver and spleen leads to organomegaly. Presence of Gaucher cells in the bone marrow and spleen 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 Medison Pharma represents exclusively Shire pharmaceuticals products in Israel in varied therapeutics fields: Hematology, Nephrology and Human Genetic Therapies (Replagal®, Elaprase®, Firazyr® and now Vpriv® for the treatment of Type 1 Gaucher Disease).
For further details regarding VPRIV “Patient name” program please contact Medison Pharma offices at 972-3-9250250
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