ﺑﺎﺯﮔﺸﺖ ﺑﻪ ﺻﻔﺤﻪ ﻗﺒﻠﯽ
خرید پکیج
تعداد آیتم قابل مشاهده باقیمانده : 0 مورد

Velaglucerase alfa (glucocerebrosidase): Pediatric drug information

Velaglucerase alfa (glucocerebrosidase): Pediatric drug information
2025© UpToDate, Inc. and its affiliates and/or licensors. All Rights Reserved.
ALERT: US Boxed 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 velaglucerase alfa in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (eg, anaphylaxis) occurs, discontinue velaglucerase alfa 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.

Brand Names: US
  • Vpriv
Brand Names: Canada
  • Vpriv
Therapeutic Category
  • Enzyme, Glucocerebrosidase;
  • Gaucher's Disease, Treatment Agent
Dosing: Pediatric

Dosage guidance:

Safety: Pretreatment with antihistamines and/or corticosteroids can be considered for prevention of infusion reactions in patients with previous hypersensitivity reactions to velaglucerase alfa or other enzyme replacement therapies. Initiate in a health care setting with appropriate medical monitoring and support measures, including CPR equipment, readily available during administration.

Gaucher disease, type 1, nonneurologic manifestations

Gaucher disease , type 1 , nonneurologic manifestations: Note: Efficacy of velaglucerase is limited to short and long-term improvements in skeletal deterioration, visceromegaly, and hematologic abnormalities of Gaucher disease type 1; positive effects on neurologic manifestations have not been observed (Ref).

Treatment-naive:

Infants and Children <4 years: Limited data available: IV: Usual dose range: 45 to 60 units/kg every other week; adjust based on disease activity; mean reported initial dose: 54.6 ± 23.1 units/kg (Ref).

Children ≥4 years and Adolescents: IV: 60 units/kg every other week; adjust based upon disease activity; dosing range: 15 to 60 units/kg every other week (Ref).

Conversion from imiglucerase therapy to velaglucerase:

Children ≥4 years and Adolescents: IV: Initiate velaglucerase at the same previously stable imiglucerase dose, starting 2 weeks after last imiglucerase dose; adjust based upon disease activity (Ref).

Gaucher disease, type 3, nonneurologic manifestations

Gaucher disease, type 3, nonneurologic manifestations: Limited data available: Note: Efficacy of velaglucerase is limited to short and long-term improvements in skeletal deterioration, visceromegaly, and hematologic abnormalities of Gaucher disease type 3; positive effects on neurologic manifestations have not been observed (Ref).

Infants, Children, and Adolescents: IV: 60 units/kg every other week; adjust based upon disease activity (Ref). Note: Based on experience in patients with type 1 Gaucher disease, when switching from imiglucerase to velaglucerase alfa, consider initiating velaglucerase alfa treatment 2 weeks after the last imiglucerase dose and at the same dose as stable imiglucerase therapy (Ref).

Dosing: Kidney Impairment: Pediatric

There are no dosage adjustments provided in manufacturer's labeling.

Dosing: Liver Impairment: Pediatric

There are no dosage adjustments provided in manufacturer's labeling.

Dosing: Adult

(For additional information see "Velaglucerase alfa (glucocerebrosidase): Drug information")

Dosage guidance:

Clinical considerations: Pretreatment with antihistamines and/or corticosteroids can be considered for prevention of subsequent infusion reactions in patients with previous hypersensitivity reactions to velaglucerase alfa or other enzyme replacement therapies; during clinical studies, patients were not routinely premedicated prior to infusion.

Gaucher disease

Gaucher disease (type 1): IV: 60 units/kg administered every 2 weeks; adjust dose based upon disease activity (range: 15 to 60 units/kg evaluated in clinical trials).

Note: When switching from imiglucerase to velaglucerase alfa in stable patients, initiate treatment 2 weeks after the last imiglucerase dose and at the same dose.

Dosing: Kidney Impairment: Adult

No dosage adjustment provided in manufacturer's labeling.

Dosing: Liver Impairment: Adult

No dosage adjustment provided in manufacturer's labeling.

Adverse Reactions

The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.

>10%:

Gastrointestinal: Abdominal pain (15% to 19%)

Hematologic & oncologic: Prolonged partial thromboplastin time (5% to 11%; more common in children and adolescents)

Hypersensitivity: Hypersensitivity reaction (23% to 52%; including anaphylaxis: <1%)

Nervous system: Dizziness (8% to 22%), fatigue (≤15%), headache (30% to 35%)

Neuromuscular & skeletal: Arthralgia (8% to 15%; knee), asthenia (≤15%), back pain (17% to 18%)

Miscellaneous: Fever (13% to 22%; more common in children and adolescents)

1% to 10%:

Cardiovascular: Flushing (>2%), hypertension (>2%), hypotension (>2%), tachycardia (>2%)

Dermatologic: Skin rash (>2%; more common in children and adolescents), urticaria (>2%)

Gastrointestinal: Nausea (6% to 10%)

Immunologic: Antibody development (neutralizing: 2%)

Neuromuscular & skeletal: Ostealgia (>2%)

Postmarketing: Gastrointestinal: Vomiting

Contraindications

There are no contraindications listed in the US manufacturer's labeling.

Canadian labeling: Hypersensitivity to velaglucerase alfa or any component of the formulation.

Warnings/Precautions

Concerns related to adverse effects:

• Antibody formation: The development of IgG antibodies has been reported; the clinical significance is unknown. Patients with an immune response to other enzyme replacement therapies who are switching to velaglucerase alfa should be monitored for antibody development.

• Hypersensitivity reactions: Velaglucerase alfa may cause severe hypersensitivity reactions, including anaphylaxis (sometimes life-threatening). The most common hypersensitivity reactions reported in clinical trials include asthenia, dizziness, fatigue, fever, headache, hyper-/hypotension, nausea, and pyrexia. Most reactions were mild and occurred during the first 6 months of treatment. Management strategies of more severe reactions include symptomatic treatment, pretreatment with antihistamines, antipyretics, and/or corticosteroids, and slowing of the infusion rate. Infusion should be discontinued if anaphylaxis or other acute reactions occur.

Warnings: Additional Pediatric Considerations

Pediatric patients (4 to 17 years) may experience a higher frequency of some adverse effects than adults (>10% difference in incidence), including fever, rash, and prolonged aPTT.

Dosage Forms: US

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution Reconstituted, Intravenous [preservative free]:

Vpriv: 400 units (1 ea)

Generic Equivalent Available: US

No

Pricing: US

Solution (reconstituted) (Vpriv Intravenous)

400 unit (per each): $1,779.32

Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.

Dosage Forms: Canada

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution Reconstituted, Intravenous:

Vpriv: 400 units (1 ea)

Administration: Pediatric

Note: Monitor patients closely for infusion-related or hypersensitivity reactions. Initiate infusion in a health care setting that includes cardiopulmonary monitoring and can provide resuscitation if necessary.

Parenteral: IV: Infuse over 60 minutes through an in-line low protein-binding 0.2 or 0.22 micron filter. Do not infuse other products in the same infusion tubing.

Rate adjustment for hypersensitivity:

Mild to moderate hypersensitivity reactions: Treatment of hypersensitivity reactions should be based on the severity of the reaction and may include temporarily interrupting the infusion, decreasing the infusion rate, and/or administration of antihistamines, antipyretics, and/or corticosteroids (Ref). Consider premedication with antihistamines and/or corticosteroids for subsequent infusions and reduction of infusion rate; closely monitor when readministering.

Anaphylaxis or severe hypersensitivity: Discontinue therapy immediately for severe hypersensitivity reactions or anaphylaxis and treat appropriately. If the decision is made to readminister after anaphylaxis, consider premedication with antihistamines and/or corticosteroids for subsequent infusions and reduction of infusion rate; appropriately trained personnel and emergency medications should be readily available. Monitor patient closely for recurrence.

Administration: Adult

IV: Infuse over 1 hour; use an inline, low protein-binding 0.2- or 0.22-micron filter during infusion. Do not infuse other products in the same infusion tubing.

Storage/Stability

Store intact vials at 2°C to 8°C (36°F to 46°F). Once reconstituted, the product should be used immediately. If immediate use is not possible, the reconstituted or diluted product may be stored for up to 24 hours at 2°C to 8°C (36°F to 46°F). The infusion should be completed within 24 hours of reconstitution. Do not freeze. Protect from light. Discard any unused solution.

Use

Long-term enzyme replacement therapy for patients with type 1 Gaucher disease (FDA approved in ages ≥4 years and adults); has also been used for patients with type 3 Gaucher disease.

Metabolism/Transport Effects

None known.

Drug Interactions

Note: Interacting drugs may not be individually listed below if they are part of a group interaction (eg, individual drugs within “CYP3A4 Inducers [Strong]” are NOT listed). For a complete list of drug interactions by individual drug name and detailed management recommendations, use the drug interactions program

There are no known significant interactions.

Pregnancy Considerations

Based on available information, an increased risk of adverse pregnancy outcomes has not been observed following maternal use of velaglucerase alfa (Elstein 2014; Lau 2018). Pregnancy may exacerbate existing type I Gaucher disease or result in new symptoms. Women with type I Gaucher disease have an increased risk of spontaneous abortion if disease is not well controlled. Adverse pregnancy outcomes, including hepatosplenomegaly and thrombocytopenia, may occur.

Monitoring Parameters

Signs and symptoms of anaphylaxis and hypersensitivity; CBC, liver enzymes, ECG and echocardiogram (baseline, every 12 months until clinical goals achieved, then every 12 to 24 months) (Baldellou 2004; Charrow 2004), IgG antibodies to velaglucerase (baseline; particularly in patients with a history of antibody formation to other enzyme replacement therapies); bone density studies.

Mechanism of Action

Velaglucerase alfa, which contains the same amino acid sequence as endogenous glucocerebrosidase, catalyzes the hydrolysis of glucocerebroside to glucose and ceramide in the lysosome. In patients with type 1 Gaucher’s disease, glucocerebrosidase deficiency results in accumulation of glucocerebroside in macrophages, thereby causing the associated signs and symptoms. Velaglucerase alfa is used to diminish hepatosplenomegaly and improve anemia, thrombocytopenia, and bone disease.

Pharmacokinetics (Adult Data Unless Noted)

Note: Values reported below based on combined pediatric patient (4-17 years) and adult data.

Distribution: Vd: Steady-state: 0.08-.0.11 L/kg

Half-life elimination: 11-12 minutes

Brand Names: International
International Brand Names by Country
For country code abbreviations (show table)

  • (AR) Argentina: Vpriv;
  • (AT) Austria: Vpriv;
  • (AU) Australia: Vpriv;
  • (BE) Belgium: Vpriv;
  • (BG) Bulgaria: Vpriv;
  • (BR) Brazil: Vpriv;
  • (CH) Switzerland: Vpriv;
  • (CO) Colombia: Vpriv;
  • (CZ) Czech Republic: Vpriv;
  • (DE) Germany: Vpriv;
  • (EE) Estonia: Vpriv;
  • (ES) Spain: Vpriv;
  • (FI) Finland: Vpriv;
  • (FR) France: Vpriv;
  • (GB) United Kingdom: Vpriv;
  • (GR) Greece: Vpriv;
  • (HR) Croatia: Vpriv;
  • (HU) Hungary: Vpriv;
  • (IE) Ireland: Vpriv;
  • (IT) Italy: Vpriv;
  • (JP) Japan: Vpriv;
  • (KR) Korea, Republic of: Vpriv;
  • (LV) Latvia: Vpriv;
  • (MX) Mexico: Vpriv;
  • (NL) Netherlands: Vpriv;
  • (NO) Norway: Vpriv;
  • (PR) Puerto Rico: Vpriv;
  • (PT) Portugal: Vpriv;
  • (QA) Qatar: Vpriv;
  • (RO) Romania: Vpriv;
  • (RU) Russian Federation: Vpriv;
  • (SA) Saudi Arabia: Vpriv;
  • (SE) Sweden: Vpriv;
  • (SI) Slovenia: Vpriv;
  • (SK) Slovakia: Vpriv;
  • (TR) Turkey: Vpriv;
  • (TW) Taiwan: Vpriv;
  • (UA) Ukraine: Vpriv;
  • (UY) Uruguay: Vpriv;
  • (ZA) South Africa: Vpriv
  1. Baldellou A, Andria G, Campbell PE, et al. Paediatric non-neuronopathic Gaucher disease: recommendations for treatment and monitoring. Eur J Pediatr. 2004;163(2):67-75. [PubMed 14677062]
  2. Charrow J, Andersson HC, Kaplan P, et al. Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations. J Pediatr. 2004;144(1):112-120. [PubMed 14722528]
  3. Deegan P, Lau H, Elstein D, et al. Long-term treatment of Gaucher disease with velaglucerase alfa in ERT-naïve patients from the Gaucher Outcome Survey (GOS) registry. J Clin Med. 2024;13(10):2782. doi:10.3390/jcm13102782 [PubMed 38792324]
  4. Elstein D, Hughes D, Goker-Alpan O, et al. Outcome of pregnancies in women receiving velaglucerase alfa for Gaucher disease [published correction appears in J Obstet Gynaecol Res. 2014;40(9):2088]. J Obstet Gynaecol Res. 2014;40(4):968-975. doi: 10.1111/jog.12254. [PubMed 24612151]
  5. Elstein D, Mellgard B, Dinh Q, et al. Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease: Data from phase 3 clinical trials. Mol Genet Metab. 2017;122(1-2):113-120. [PubMed 28851512]
  6. Jmoudiak M, Futerman AH. Gaucher disease: pathological mechanisms and modern management. Br J Haematol. 2005;129(2):178-188. [PubMed 15813845]
  7. Kaplan P, Baris H, De Meirleir L, et al. Revised recommendations for the management of Gaucher disease in children. Eur J Pediatr. 2013;172(4):447-458. doi:10.1007/s00431-012-1771-z [PubMed 22772880]
  8. Lau H, Belmatoug N, Deegan P, et al. Reported outcomes of 453 pregnancies in patients with Gaucher disease: an analysis from the Gaucher outcome survey. Blood Cells Mol Dis. 2018;68:226-231. doi: 10.1016/j.bcmd.2016.10.003. [PubMed 27839985]
  9. Pastores GM, Weinreb NJ, Aerts H, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol. 2004;41(4 Suppl 5):4-14. [PubMed 15468045]
  10. Refer to manufacturer's labeling.
  11. Vellodi A, Tylki-Szymanska A, Davies EH, et al. Management of neuronopathic Gaucher disease: revised recommendations. J Inherit Metab Dis. 2009;32(5):660-664. doi:10.1007/s10545-009-1164-2 [PubMed 19655269]
  12. Vpriv (velaglucerase alfa) [prescribing information]. Lexington, MA: Takeda Pharmaceuticals USA Inc; July 2024.
  13. Vpriv (velaglucerase alfa) [product monograph]. Toronto, Ontario, Canada: Takeda Canada Inc; received December 2020.
  14. Weinreb NJ, Aggio MC, Andersson HC, et al; International Collaborative Gaucher Group (ICGG). Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients. Semin Hematol. 2004;41(4 Suppl 5):15-22. [PubMed 15468046]
Topic 15623 Version 137.0