Chemical Name: Ceramidase, glucosyl-(human HT-1080 cell)
Molecular Formula: C2532H3854N672O711S16
CAS Number: 0884604-91-5
Uses for Velaglucerase Alfa
Velaglucerase Alfa Dosage and Administration
Administer under the supervision of a clinician.1
For solution compatibility information, see Compatibility under Stability.
Administer by IV infusion.1
Administer using an inline, low-protein-binding, 0.2-mcm particulate filter.1
Determine number of vials to be reconstituted based on patient weight.1
Reconstitute appropriate number of vials containing 200 or 400 units of velaglucerase alfa lyophilized powder with 2.2 or 4.3 mL of sterile water for injection, respectively, to provide a solution containing 100 units/mL.1 Mix gently; do not shake vial.1
Rate of Administration
Administer over 1 hour.1
Children and adolescents 4–17 years of age: Initially, 60 units/kg every 2 weeks.1
Adjust dosage based on achievement and maintenance of 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
Initially, 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
No known contraindications.1
Hypersensitivity reactions reported.1
Carefully reevaluate therapy if substantial clinical evidence of hypersensitivity develops.1 Treat severe hypersensitivity reactions in accordance with current emergency practice standards; appropriate medical support should be readily available.1
Use with caution in patients who have exhibited manifestations of hypersensitivity reactions to velaglucerase alfa, any ingredient in the formulation, or other enzyme replacement therapy.1
Infusion-related reactions (e.g., headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, pyrexia) reported.1
Depending on severity, manage infusion-related reactions by slowing the infusion rate, providing appropriate medical treatment (e.g., antihistamines, antipyretics, and/or corticosteroids), and/or interrupting and then resuming treatment at a slower infusion rate.1
Pretreatment with antihistamines and/or corticosteroids may prevent subsequent reactions in patients who have experienced infusion-related reactions requiring treatment.1
Continue to monitor for antibodies in patients with an immune response to other enzyme replacement therapies who are switched to velaglucerase alfa.1
Safety and efficacy not established in children <4 years of age.1
Insufficient experience in patients ≥65 years of age to determine whether geriatric patients respond differently than younger adults; select dosage with caution.1
Common Adverse Effects
Infusion-related reactions, headache, upper respiratory tract infection, dizziness, pyrexia, abdominal pain, back pain, joint (knee) pain, asthenia/fatigue, prolonged aPTT, nausea.1
Upper respiratory tract infection, rash, prolonged aPTT, and pyrexia reported more frequently in pediatric patients than in adults.1
Interactions for Velaglucerase Alfa
Formal drug interaction studies not performed to date.1
Velaglucerase Alfa Pharmacokinetics
Accumulation not observed in patients receiving 60 units/kg every 2 weeks for 37 weeks.1
Not known whether velaglucerase alfa distributes into milk.1
Powder for Injection
Following reconstitution or dilution, 2–8°C for up to 24 hours; protect from light; do not freeze.1
For information on systemic interactions resulting from concomitant use, see Interactions.
Sodium chloride 0.9%
Enzyme replacement therapy for type 1 Gaucher's disease increases the degradation of glucosylceramide (glucocerebroside) in macrophages1 3 4 5 7 8 10 26 27 by hydrolyzing the β-glycoside linkage of glucocerebroside to glucose and ceramide (N-acylsphingosine) 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 6 7 8 9 10 11 12 13 14 15 16 17 18 19
Advice to Patients
Risk of hypersensitivity and infusion-related reactions.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. (See Cautions.)1
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.
For injection, for IV infusion
AHFS DI Essentials. © Copyright, 2016, American Society of Health-System Pharmacists, Inc., 7272 Wisconsin Avenue, Bethesda, Maryland 20814.
Date published: February 15, 2013
Last reviewed: February 15, 2013
Date modified: February 08, 2016
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11. Reviewers’ comments (personal observations) on alglucerase.
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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. [PubMed 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. [IDIS 289025] [PubMed 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. [IDIS 289028] [PubMed 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. [PubMed 269414]
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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. [PubMed 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. [IDIS 280934] [PubMed 2023606]
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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. [PubMed 1951303]
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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. [PubMed 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. [PubMed 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. [PubMed 22047948]
36. US Food and Drug Administration. Center for Drug Evaluation and Research. Application number 22-575: Chemistry review(s). From FDA website.
More about velaglucerase alfa
- Other brands: VPRIV