Velaglucerase Alfa

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. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide.

• VPRIV is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

• None.contraindicated

Administration of VPRIV should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis ( 2.1 ) Recommended Starting Dose in Adults and Pediatric Patients 4 Years of Age or Older: Patients Naïve to Enzyme Replacement Therapy: 60 Units/kg ( 2.2 ) Patients being treated with stable imiglucerase dosages for Gaucher disease: Can switch to VPRIV at previous imiglucerase dose two weeks after last imiglucerase dose ( 2.3 ) Determine number of vials to be reconstituted based on patient's actual weight and prescribed dose ( 2.4 ) Supplied VPRIV lyophilized powder must be reconstituted with Sterile Water for Injection ( 2.4 ) Reconstituted VPRIV solution must be diluted in 100 mL of 0.9% Sodium Chloride Injection prior to intravenous infusion ( 2.4 ) Administer the diluted VPRIV solution through an in-line low protein-binding 0.2 or 0.22 µm filter ( 2.5 ) 2.1 Recommendations Prior to VPRIV treatment Administration of VPRIV should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] . Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1)] .

See boxed warning ( 5.1 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have occurred in patients treated with enzyme replacement therapies, including VPRIV. VPRIV-treated patients have had these reactions occur in clinical studies and postmarketing experience [see Adverse Reactions (6.1) and Clinical studies (14) ] . Hypersensitivity reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. Patients were not routinely pre-medicated prior to infusion of VPRIV during clinical studies. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia/body temperature increased. Generally the reactions were mild and, in treatment-naïve patients, onset occurred mostly during the first 6 months of treatment and tended to occur less frequently with time. Additional hypersensitivity reactions of chest discomfort, dyspnea, pruritus and vomiting have been reported in post-marketing experience. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Administration of VPRIV should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.

Most common adverse reactions (≥10%) are: hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, prolonged activated PTT, fatigue/asthenia, and pyrexia ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Takeda Pharmaceuticals at 1-800-828-2088 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The data described below reflect exposure of 94 patients with type 1 Gaucher disease who received VPRIV at doses ranging from 15 Units/kg to 60 Units/kg every other week in 5 clinical studies. Fifty-four (54) patients were naïve to enzyme replacement therapy (ERT) and received VPRIV for 9 months and 40 patients switched from imiglucerase to VPRIV treatment and received VPRIV for 12 months [see Clinical Studies (14) ] . Patients were between 4 and 71 years old at time of first treatment with VPRIV, and included 46 male and 48 female patients. The most serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions [see Warnings and Precautions (5.1) ] . The most commonly reported adverse reactions (occurring in ≥10% of patients) that were considered related to VPRIV are shown in Table 1. The most common adverse reactions were hypersensitivity reactions.

8.1 Pregnancy Risk Summary Available data on use of velaglucerase alfa in pregnant women includes more than 300 pregnancies reported from the pharmacovigilance database and published observational cohort studies, including the international Gaucher Disease registry. While available data cannot definitively establish or exclude the absence of a velaglucerase alfa associated risk during pregnancy, these data have not identified an association with use of velaglucerase alfa during pregnancy and major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproduction studies no fetal harm was observed in rats or rabbits when velaglucerase alfa was administered intravenously during organogenesis at doses with exposures up to 1.8 times and 4.3 times, respectively, the recommended human daily dose (see Data ) . The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of major birth defects, loss, and other adverse outcomes. In the U.S. general population, the estimated major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.

Metabolism

In a multicenter study conducted in pediatric (N=7, 4 to 17 years old) and adult (N=15, 19 to 62 years old) patients with type 1 Gaucher disease, pharmacokinetic evaluations were performed at Weeks 1 and 37 following 60-minute intravenous infusions of VPRIV 60 Units/kg every other week. Serum velaglucerase alfa concentrations declined rapidly with a mean half life of 11 to 12 minutes.

• Feb 26, 2010BLABLA022575Shire Human Genetic

1. 1Inappropriate Schedule Of Product Administration21010%
2. 2Product Dose Omission Issue1989.8%
3. 3Covid-191678.2%
4. 4Fatigue1527.5%
5. 5Death1246.1%
6. 6Fall1125.5%
7. 7Pyrexia1115.5%
8. 8Malaise1105.4%
9. 9Headache1055.2%
10. 10Weight Increased1035.1%
11. 11Infusion Related Reaction1025.0%
12. 12Nausea954.7%
13. 13Arthralgia944.6%
14. 14Weight Decreased904.4%
15. 15Abdominal Pain894.4%

The 10 most recently updated of 23 ClinicalTrials.gov registrations naming Velaglucerase Alfa as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
  Not yet recruiting · Observational · 60 enrolled · Takeda
  NCT05669729updated 2026-04-03
• Survey Study for Velaglucerase Alfa (VPRIV) in Japan
  Completed · Observational · 63 enrolled · Takeda
  NCT03625882updated 2026-03-04
• A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
  Completed · Phase 3 · Interventional · 20 enrolled · Takeda
  NCT05529992updated 2025-06-26
• A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
  Completed · Observational · 11 enrolled · Takeda
  NCT04721366updated 2024-03-29
• A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
  Terminated · Observational · 2 enrolled · Shire
  NCT04094181updated 2024-02-02
• A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
  Completed · Phase 4 · Interventional · 4 enrolled · Takeda
  NCT04718779updated 2023-12-21
• An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3
  Withdrawn · Phase 2 · Phase 3 · Interventional · 0 enrolled · AVROBIO
  NCT05815004updated 2023-08-09
• Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India
  Completed · Observational · 21 enrolled · Shire
  NCT04429984updated 2023-06-07
• Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
  Completed · Phase 4 · Interventional · 15 enrolled · Shaare Zedek Medical Center
  NCT04120506updated 2022-10-28
• Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
  Completed · Phase 4 · Interventional · 21 enrolled · Shire
  NCT02574286updated 2022-02-01

CPIC-curated drug–gene pairs for Velaglucerase Alfa. Levels describe the strength of curated evidence and guideline status — never a recommendation to test or to adjust therapy.

• GBACPIC A/B (provisional)

Frequently asked questions

How does Velaglucerase Alfa work?

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. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide.

What is Velaglucerase Alfa used for?

According to FDA labeling, Velaglucerase Alfa carries indications including: VPRIV is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Velaglucerase Alfa?

Velaglucerase Alfa is classified as Enzymes, Hydrolytic Lysosomal Glucocerebroside-specific Enzyme, Enzymatic Activity, Lipid Metabolism Alteration.

What are the brand names for Velaglucerase Alfa?

Velaglucerase Alfa is marketed under brand names including Vpriv.

What are the contraindications for Velaglucerase Alfa?

Velaglucerase Alfa labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.