March 1, 2010 — The US Food and Drug Administration (FDA) has approved velaglucerase alfa injection (VPRIV, Shire Human Genetic Therapies, Inc), a hydrolytic lysosomal glucocerebroside-specific enzyme for the long-term treatment of type 1 Gaucher disease in pediatric and adult patients.
Gaucher disease is a chronic, progressive, autosomal-recessive inherited metabolic disorder that affects 1 in 50,000 to 100,000 people. Type 1 is the most common (90%) form of the condition and occurs with increased prevalence (1 in 500 - 1000) among Jews of Ashkenazi (European) descent.
Patients with Gaucher disease are deficient in glucocerebrosidase, leading to the accumulation of the cell membrane lipid glucocerebroside. Subsequent build-ups in the liver and spleen lead to organomegaly; accretions in the bone marrow and spleen often result in clinically significant anemia and thrombocytopenia.
FDA approval of velaglucerase replacement therapy was based on a priority review of data from 3 clinical studies of 82 patients aged 4 years and older, some of whom switched from imiglucerase therapy.
"The approval of [velaglucerase] will provide a safe and effective alternative treatment for patients with Gaucher disease," said Julie Beitz, MD, director of the FDA's Office of Drug Evaluation III, in an agency news release. "Patients who previously received [imiglucerase] as an enzyme replacement therapy for their Type 1 Gaucher disease can be safely switched to VPRIV." Imiglucerase (Cerezyme, Genzyme Corp) is currently in short supply, FDA officials noted.
The recommended velaglucerase regimen is 60 IU/kg administered every other week as a 1-hour intravenous infusion. No dosing changes are required for stable patients switching from imiglucerase; for others, individualized dosing adjustments should be based on achievement and maintenance of therapeutic goals.
The most commonly reported adverse events with velaglucerase in clinical trials were infusion related and included symptoms such as headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia. Infusion reactions were generally mild and occurred most frequently during the first 6 months of therapy in treatment-naive patients.
Pediatric patients were more likely than adults (>10% difference) to experience rash, upper respiratory tract infection, prolonged partial thromboplastin time, and pyrexia.
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Cite this: FDA Approves Velaglucerase Infusion for Gaucher Disease - Medscape - Mar 01, 2010.
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