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. First approved in 2010.
Drug data last refreshed 11h ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
VPRIV (velaglucerase alfa) is a recombinant lysosomal enzyme replacement therapy for Gaucher disease, a rare lysosomal storage disorder caused by deficiency of beta-glucocerebrosidase. The drug catalyzes the hydrolysis of accumulated glucocerebroside in macrophages, reducing organomegaly, anemia, and thrombocytopenia. It is administered intravenously and addresses a critical unmet need in a small patient population with life-limiting complications.
$0.009BPart D
LOE ApproachingProduct is approaching loss of exclusivity with modest Part D spending of $9M across 253 claims in 2023, suggesting a stable but small commercial footprint requiring defensive strategy.
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
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (14)
A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
Started Jan 2027
60 enrolled
Gaucher Disease
A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
Started Jan 2023
20 enrolled
Gaucher Disease
Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India
Started Jul 2021
21 enrolled
Gaucher Disease
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
Started Apr 2021
4 enrolled
Gaucher Disease
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
Started Jan 2021
11 enrolled
Gaucher Disease
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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.