Velaglucerase Alfa (Monograph)

Brand name: VPRIV
Drug class: Enzymes
Chemical name: Ceramidase, glucosyl-(human HT-1080 cell)
Molecular formula: C₂₅₃₂H₃₈₅₄N₆₇₂O₇₁₁S₁₆
CAS number: 0884604-91-5

Medically reviewed by Drugs.com on Mar 12, 2025. Written by ASHP.

Introduction

Biosynthetic (gene activation technology origin) form of human β-glucocerebrosidase (glucosylceramidase).¹ ³ ⁴ ³³

Uses for Velaglucerase Alfa

Gaucher Disease

Long-term enzyme replacement therapy in patients with nonneuronopathic (type 1) Gaucher disease¹ ⁵ ³² ³³ (designated an orphan drug by FDA for this use).²

Velaglucerase Alfa Dosage and Administration

General

Administer under the supervision of a clinician.¹

Administration

IV Administration

For solution compatibility information, see Compatibility under Stability.

Administer by IV infusion.¹

Administer using an inline, low-protein-binding, 0.2-µm particulate filter.¹

Velaglucerase alfa does not contain preservatives; prepare solutions immediately before use.¹ If immediate use is not possible, complete infusion within 24 hours of reconstitution.¹

Reconstitution

Determine number of vials to be reconstituted based on patient weight.¹

Reconstitute appropriate number of vials containing 400 units of velaglucerase alfa lyophilized powder with 4.3 mL of sterile water for injection, respectively, to provide a solution containing 100 units/mL.¹ Mix gently; do not shake.¹

Dilution

Withdraw appropriate dose from reconstituted vials and dilute with 100 mL of 0.9% sodium chloride injection.¹ Mix solution gently; do not shake.¹

Rate of Administration

Administer over 1 hour.¹

Dosage

Pediatric Patients

Gaucher Disease

IV

Children and adolescents ≥4 years of age: 60 units/kg every 2 weeks.¹

Adjust dosage based on achievement and maintenance of patient's therapeutic goals.¹

Patients receiving imiglucerase can be switched to velaglucerase alfa at an equivalent dosage.¹

Adults

Gaucher Disease

IV

60 units/kg every 2 weeks.¹

Adjust dosage based on achievement and maintenance of the patient's therapeutic goals; dosages ranging from 15–60 units/kg every 2 weeks were evaluated in clinical trials.¹

Patients receiving imiglucerase can be switched to velaglucerase alfa at an equivalent dosage.¹

Cautions for Velaglucerase Alfa

Contraindications

No known contraindications.¹

Warnings/Precautions

Sensitivity Reactions

Hypersensitivity Reactions

Hypersensitivity reactions, including anaphylaxis, reported.¹

Manage hypersensitivity reactions based on severity.¹ If anaphylaxis or other severe hypersensitivity occurs, discontinue velaglucerase alfa immediately and initiate appropriate therapy.¹ For mild reactions, may reduce infusion rate, temporarily interrupt infusion, and/or administer antihistamines, antipyretics, and/or corticosteroids.¹ Ensure that appropriate medical support is readily available during administration.¹

Premedication with antihistamines and/or corticosteroids may prevent subsequent reactions in patients who have experienced a reaction to velaglucerase alfa or other enzyme replacement therapy.¹

Antibody Formation

As with all therapeutic proteins, there is a potential for immunogenicity with velaglucerase alfa therapy.¹ Development of IgG antibodies to velaglucerase alfa reported in at least one patient.¹ Relationship between such antibodies and hypersensitivity risk not known.¹

Continue to monitor for antibodies in patients with an immune response to other enzyme replacement therapies who are switched to velaglucerase alfa.¹

Specific Populations

Pregnancy

Category B.¹ No adequate and well-controlled studies in pregnant women; use during pregnancy only if clearly needed.¹ Animal studies suggest no evidence of fetal harm.¹

Lactation

Not known whether velaglucerase alfa is distributed into milk.¹ Use with caution.¹ Consider the known benefits of breast-feeding along with the woman's clinical need for velaglucerase alfa and any potential adverse effects of the drug or disease on the infant.¹

Pediatric Use

Safety and efficacy not established in children <4 years of age.¹

Geriatric Use

Clinical response and adverse effects in geriatric patients appear to be similar to those of younger adults; in general, select dosage with caution in geriatric patients.¹

Common Adverse Effects

Hypersensitivity reactions, headache, dizziness, pyrexia, abdominal pain, back pain, joint pain, asthenia/fatigue, prolonged aPTT, nausea.¹

Drug Interactions

Formal drug interaction studies not performed to date.¹

Velaglucerase Alfa Pharmacokinetics

Absorption

Bioavailability

Accumulation not observed in patients receiving 60 units/kg every 2 weeks for 37 weeks.¹

Distribution

Extent

Not known whether velaglucerase alfa distributes into milk.¹

Elimination

Half-life

11–12 minutes.¹

Stability

Storage

Parenteral

Powder for Injection

2–8°C; protect from light.¹ Do not freeze.¹

Following reconstitution or dilution, 2–8°C for up to 24 hours; protect from light; do not freeze.¹

Compatibility

Parenteral

Solution Compatibility1

Compatible
Sodium chloride 0.9%

Actions

Replaces the deficient endogenous enzyme (glucocerebrosidase) in patients with Gaucher disease.¹ ³ ⁴
Gaucher disease is an autosomal recessive lysosomal storage disorder characterized by a deficiency of the enzyme glucocerebrosidase, which results in accumulation of glucocerebroside within the lysosomes of macrophages in the liver, spleen, bone marrow, and other organs; clinical manifestations include hepatosplenomegaly, anemia, thrombocytopenia, and skeletal complications (e.g., osteopenia, osteonecrosis, progressive joint destruction, fractures).¹ ³ ⁴ ¹³ ³⁴
Enzyme replacement therapy in patients with type 1 Gaucher disease increases the degradation of glucocerebroside to glucose and ceramide, with resultant reduction in liver and spleen size, amelioration of anemia and thrombocytopenia, decreased bone pain, decreased cachexia, and increased bone remineralization over a period of several years.⁴ ⁷ ⁸ ⁹ ¹² ¹³ ¹⁴ ¹⁵ ¹⁶ ¹⁷ ¹⁸ ¹⁹

Advice to Patients

Risk of hypersensitivity reactions, including anaphylaxis.¹ Advise patients of the possible signs and symptoms of a hypersensitivity reaction and to seek immediate medical care if such manifestations occur.¹
Importance of informing clinicians of existing or contemplated concomitant therapy, including prescription and OTC drugs and herbal supplements, as well as any concomitant illnesses.¹
Importance of women informing clinicians if they are or plan to become pregnant or plan to breast-feed.¹
Importance of informing patients of other important precautionary information.¹ (See Cautions.)

Preparations

Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.

Velaglucerase Alfa
Routes	Dosage Forms	Strengths	Brand Names	Manufacturer
Parenteral	For injection, for IV infusion	400 units	VPRIV	Shire

References

1. Shire Human Genetic Therapies, Inc. VPRIV (velaglucerase alfa for injection) prescribing information. Cambridge, MA; 2015 April.

2. Food and Drug Administration. Cumulative list of orphan drugs designated and/or approved. Rockville, MD; 2009 Jun 8. Accessed 2011 Dec 14. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/default.htm

3. Burrow TA, Grabowski GA. Velaglucerase alfa in the treatment of Gaucher disease type 1. Clin Invest. 2011; 1:285-93.

4. Cox TM. Gaucher disease: clinical profile and therapeutic developments. Biologics. 2010; 4:299-313. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3010821/ https://pubmed.ncbi.nlm.nih.gov/21209725

5. Zimran A, Altarescu G, Philips M et al. Phase 1/2 and extension study of velaglucerase alfa and replacement therapy in adults with type 1 Gaucher disease: 48-month experience. Blood. 2010; 115:4651-6. https://pubmed.ncbi.nlm.nih.gov/20299511

6. Beutler E. Gaucher's disease. N Engl J Med. 1991; 325:1354-60. https://pubmed.ncbi.nlm.nih.gov/1922238

7. Genzyme Corporation. Cerezyme (imiglucerase) for injection prescribing information. Cambridge, MA; 2003 Mar.

8. Barton NW, Furbish FS, Murray GJ et al. Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease. Proc Natl Acad Sci USA. 1990; 87:1913-6. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC53594/ https://pubmed.ncbi.nlm.nih.gov/2308952

9. Anon. Alglucerase for Gaucher’s disease. Med Lett Drugs Ther. 1991; 33:82. https://pubmed.ncbi.nlm.nih.gov/1865852

12. Weinreb NJ, Charrow J, Andersson HC et al. Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Register. Am J Med. 2002; 113:112-9. https://pubmed.ncbi.nlm.nih.gov/12133749

13. Niederau C, Haussinger D. Gaucher’s disease: a review for the internist and hepatologist. Hepatogastroenterology. 2000; 47:984-7. https://pubmed.ncbi.nlm.nih.gov/11020862

14. Charrow J, Andersson HC, Kaplan P et al. Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: Consensus recommendations. J Pediatrics. 2003; 144:112-20.

15. Rosenthal DI, Doppelt SH, Mankin HL et al. Enzyme therapy for Gaucher disease: skeletal responses to macrophage-targeted glucocerebrosidase. Pediatrics. 1995; 96:629-37. https://pubmed.ncbi.nlm.nih.gov/7567322

16. Grabowski GA, Barton NW, Pastores G et al. Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase form natural and recombinant sources. Ann Intern Med. 1995; 122:33-9. https://pubmed.ncbi.nlm.nih.gov/7985893

17. Actelion Pharmaceuticals. Zavesca (miglustat) capsules prescribing information. South San Francisco, CA; 2003 Jul 31.

18. Elstein D, Abrahamov A, Hadas-Halpern I et al. Low-dose low-frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type I Gaucher disease. QJM. 1998; 91:483-8. https://pubmed.ncbi.nlm.nih.gov/9797931

19. Poll LW, Maas M, Terk MR et al. Response of Gaucher bone disease to enzyme replacement therapy. Br J Radiol. 2002; 75(Suppl 1):A25-36.

20. Genzyme Corporation, Cambridge, MA: Personal communication.

21. Stahl PD, Rodman JS, Miller MJ et al. Evidence for receptor-mediated binding of glycoproteins, glycoconjugates, and lysosomal glycosidases by alveolar macrophages. Proc Natl Acad Sci USA. 1978; 75:1399-1403. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC411479/ https://pubmed.ncbi.nlm.nih.gov/274729

22. Beutler E, Kay AC, Saven A et al. Enzyme-replacement therapy for Gaucher’s disease. N Engl J Med. 1991; 325:1809-10. https://pubmed.ncbi.nlm.nih.gov/1944489

23. Zimran A, Hadas-Halpern I, Abrahamov A et al. Enzyme-replacement therapy for Gaucher’s disease. N Engl J Med. 1991; 325:1810-1.

24. Barton NW, Brady RO, Murray GJ et al. Enzyme-replacement therapy for Gaucher’s disease. N Engl J Med. 1991; 325:1811. https://pubmed.ncbi.nlm.nih.gov/1944490

25. Furbish FS, Blair HE, Shiloach J et al. Enzyme replacement therapy in Gaucher’s disease: large-scale purification of glucocerebrosidase suitable for human administration. Proc Natl Acad Sci USA. 1977; 74:3560-3. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC431631/ https://pubmed.ncbi.nlm.nih.gov/269414

26. Brady RO, Kanfer JN, Shapiro D. Metabolism of glucocerebrosides: II. Evidence of an enzymatic deficiency in Gaucher’s disease. Biochem Biophys Res Commun. 1965; 18:221-5. https://pubmed.ncbi.nlm.nih.gov/14282020

27. Basu A, Prence E, Garrett K et al. Comparison of N-acyl phosphatidylethanolamines with different N-acyl groups as activators of glucocerebrosidase in various forms of Gaucher’s disease. Arch Biochem Biophys. 1985; 243:28-34. https://pubmed.ncbi.nlm.nih.gov/3933429

28. Barton NW, Brady RO, Dambrosia JM et al. Replacement therapy for inherited enzyme deficiency—macrophage-targeted glucocerebrosidase for Gaucher’s disease. N Engl J Med. 1991; 324:1464-70. https://pubmed.ncbi.nlm.nih.gov/2023606

29. Beutler E, Kay A, Saven A et al. Enzyme replacement therapy for Gaucher disease. Blood. 1991; 78:1183-9. https://pubmed.ncbi.nlm.nih.gov/1878585

30. Parker RI, Barton NW, Read EJ et al. Hematologic improvement in a patient with Gaucher disease on long-term enzyme replacement therapy: evidence for decreased splenic sequestration and improved red blood cell survival. Am J Hematol. 1991; 38:130-7. https://pubmed.ncbi.nlm.nih.gov/1951303

31. Barton NW, Brady RO, Dambrosia JM et al. Dose-dependent responses to macrophage-targeted glucocerebrosidase in a child with Gaucher disease. J Pediatr. 1992; 120:277-80. https://pubmed.ncbi.nlm.nih.gov/1735829

32. Anon. Velaglucerase (Vpriv) for Gaucher's disease. Med Lett Drugs Ther. 2010; 52:36.

33. Elstein D, Cohn GM, Wang N et al. Early achievement and maintenance of the therapeutic goals using velaglucerase alfa in type 1 Gaucher disease. Blood Cells Mol Dis. 2011; 46:119-23. Epub 2010 Aug 19. https://pubmed.ncbi.nlm.nih.gov/20727796

34. Elstein D, Foldes AJ, Zahrieh D et al. Significant and continuous improvement in bone mineral density among type 1 Gaucher disease patients treated with velaglucerase alfa: 69-month experience, including dose reduction. Blood Cells Mol Dis. 2011; 47:56-61. Epub 2011 May 4. https://pubmed.ncbi.nlm.nih.gov/21536468

35. Elstein D, Altarescu G, Maayan H et al. Booster-effect with velaglucerase alfa in patients with Gaucher disease switched from long-term imiglucerase therapy: Early Access Program results from Jerusalem. Blood Cells Mol Dis. 2012; 48:45-50. Epub 2011 Nov 1. https://pubmed.ncbi.nlm.nih.gov/22047948

36. US Food and Drug Administration. Center for Drug Evaluation and Research. Application number 22-575: Chemistry review(s). From FDA website. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2010/022575s000chemr.pdf