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Vpriv (VELAGLUCERASE ALFA)
Vpriv (generic name: VELAGLUCERASE ALFA) is a Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC] Enzyme drug developed by Shire Human Genetic. It is currently FDA-approved (first approved 2010) for Chronic non-neuropathic Gaucher's disease.
VELAGLUCERASE ALFA replaces the deficient enzyme glucocerebrosidase in patients with Gaucher's disease.
Vpriv is a hydrolytic enzyme used to treat Gaucher Disease, specifically targeting the glucocerebroside enzyme. It is administered as part of standard patient care for young children with Gaucher Disease.
At a glance
| Generic name | VELAGLUCERASE ALFA |
|---|---|
| Sponsor | Shire Human Genetic |
| Drug class | Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC] |
| Modality | Enzyme |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 2010 |
Mechanism of action
Gaucher disease is an autosomal recessive disorder caused by mutations in the GBA gene, which results in 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 of macrophages, giving rise to foam cells or Gaucher cells. Velaglucerase alfa catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside. In clinical trials VPRIV reduced spleen and liver size, and improved anemia and thrombocytopenia.In this lysosomal storage disorder (LSD), clinical features are reflective of the accumulation of Gaucher cells in the liver, spleen, bone marrow, and other organs. The accumulation of Gaucher cells in the liver and spleen leads to organomegaly. Presence of Gaucher cells in the bone marrow and spleen lead to clinically
Approved indications
- Chronic non-neuropathic Gaucher's disease
Boxed warnings
- WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement and after extended duration of therapy. Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue VPRIV and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. ( 5.1 ) Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue VPRIV and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )
Common side effects
- Hypersensitivity reaction
- Headache
- Dizziness
- Pyrexia
- Abdominal pain
- Back pain
- Joint pain
- Asthenia/Fatigue
- Activated partial thromboplastin time prolonged
- Nausea
- IgG antibodies to VPRIV
- Vomiting
Key clinical trials
- Survey Study for Velaglucerase Alfa (VPRIV) in Japan
- A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease (PHASE3)
- A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
- A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
- A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
- A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy (PHASE4)
- An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 (PHASE2,PHASE3)
- Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Vpriv CI brief — competitive landscape report
- Vpriv updates RSS · CI watch RSS
- Shire Human Genetic portfolio CI
Frequently asked questions about Vpriv
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Related
- Drug class: All Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC] drugs
- Manufacturer: Shire Human Genetic — full pipeline
- Therapeutic area: All drugs in Metabolic
- Indication: Drugs for Chronic non-neuropathic Gaucher's disease
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing