BLAINTRAVENOUSINJECTABLEPriority Review
Approved
Feb 2010
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
14
Data completeness
5/8 — Partial
Priority Review
VPRIV (velaglucerase alfa) by Takeda is gene, which results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase. Approved for gaucher disease. First approved in 2010.
Drug data last refreshed 18h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
VPRIV (velaglucerase alfa) is an intravenous enzyme replacement therapy approved in February 2010 for Gaucher disease, a rare lysosomal storage disorder caused by deficiency of the glucocerebrosidase enzyme. The drug catalyzes the hydrolysis of glucocerebroside, reducing the accumulation of pathological Gaucher cells in organs including the liver, spleen, and bone marrow. Clinical benefits include reduction in organomegaly and improvement in anemia and thrombocytopenia, addressing the primary clinical manifestations of this genetic disorder.
$0.0BPart D
Mechanism of Action
gene, which results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucocerebroside primarily in the lysosomal compartment…
Pharmacologic Class:
Hydrolytic Lysosomal Glucocerebroside-specific Enzyme
Indications (1)
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.