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Taliglucerase Alfa

Class: Enzymes
Chemical Name: glucosylceramidase
Molecular Formula: C2580H3918N680O727S17
CAS Number: 37228-64-1
Brands: Elelyso

Introduction

Biosynthetic (recombinant DNA origin) form of human β-glucocerebrosidase (glucosylceramidase).1 3 4

Uses for Taliglucerase Alfa

Gaucher Disease

Long-term enzyme replacement therapy in patients with nonneuronopathic (type 1) Gaucher disease1 5 9 (designated an orphan drug by FDA for this use).2 8

Taliglucerase Alfa Dosage and Administration

General

  • Prepare and administer under 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-mcm filter.1

Contains no preservatives; use immediately following preparation.1 (See Storage under Stability.) Discard any unused portions.1

Reconstitution

Reconstitute using proper aseptic technique.1

Determine number of vials to be reconstituted based on patient’s weight and recommended dose of 60 units/kg.1

Remove required number of vials from refrigerator prior to reconstitution; do not leave vials at room temperature for >24 hours and do not heat vials (e.g., in a microwave).1

Reconstitute each vial containing 200 units of taliglucerase alfa with 5.1 mL of sterile water for injection to provide a solution containing 40 units/mL.1 Mix gently; do not shake.1

Dilution

Withdraw appropriate dose from reconstituted vials and dilute with 0.9% sodium chloride injection to yield a final volume of 100–200 mL.1 For pediatric patients, dilute to final volume of 100–200 mL.1 For adults, may dilute to final volume of 130–150 mL; however, if volume of reconstituted drug alone is equal to or greater than this amount, do not exceed final volume of 200 mL.1 Mix gently; do not shake.1

Slight flocculation (described as translucent fibers) occurs occasionally after dilution.1

Rate of Administration

Administer over 1–2 hours.1 In pediatric patients, initial infusion rate of 1 mL/minute recommended; if tolerated, may increase up to a maximum rate of 2 mL/minute.1 In adults, an initial infusion rate of 1.2 mL/minute recommended; if tolerated, may increase up to a maximum rate of 2.2 mL/minute.1 Administer total volume over a period of ≥1 hour.1

Dosage

Pediatric Patients

Gaucher Disease
IV

Pediatric patients ≥4 years of age: 60 units/kg every 2 weeks.1

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

Patients currently receiving imiglucerase can be switched to taliglucerase alfa at the same 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.1

Patients currently receiving imiglucerase can be switched to taliglucerase alfa at the same dosage.1

Special Populations

No special population dosage recommendations at this time.1

Cautions for Taliglucerase Alfa

Contraindications

  • No known contraindications.1

Warnings/Precautions

Sensitivity Reactions

Hypersensitivity

Severe hypersensitivity reactions, including anaphylaxis, reported.1 Ensure that appropriate medical support is readily available during administration.1

Manage hypersensitivity reactions based on severity.1 If anaphylaxis or other severe hypersensitivity occurs, discontinue taliglucerase 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

Consider risks and benefits of retreatment in patients who experience a severe hypersensitivity reaction.1 Premedication with antihistamines and/or corticosteroids may prevent subsequent reactions in patients who have experienced a reaction to taliglucerase alfa or other enzyme replacement therapy.1

Antibody Formation

Development of antibodies to taliglucerase alfa reported.1 7 Although hypersensitivity reactions also occurred in some patients, relationship between antibodies and hypersensitivity not fully understood.1 Neutralizing antibodies also detected in some patients; effects on therapeutic response not known.1

Monitor for presence of antitaliglucerase alfa antibodies in patients who develop hypersensitivity to taliglucerase alfa or other enzyme replacement therapies.1

Specific Populations

Pregnancy

Insufficient experience in pregnant women; animal studies have not suggested any evidence of adverse embryofetal effects.1

Lactation

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

Pediatric Use

Use in pediatric patients ≥4 years of age supported by adequate and well-controlled studies in adults.1 In general, adverse effects are similar between adults and pediatric patients (with exception of vomiting).1

Geriatric Use

Insufficient experience in patients ≥65 years of age to determine whether geriatric patients respond differently than younger adults.1

Common Adverse Effects

Treatment-naive adults: Headache, arthralgia, fatigue, nausea, dizziness, abdominal pain, pruritus, flushing, vomiting, urticaria.1

Patients who switched from imiglucerase to taliglucerase alfa: Arthralgia, headache, extremity pain.1

Interactions for Taliglucerase Alfa

Formal drug interaction studies not performed to date.8

Taliglucerase Alfa Pharmacokinetics

Absorption

Bioavailability

Nonlinear pharmacokinetics; greater than dose-proportional increase in exposure observed at doses studied.1

No substantial accumulation or change in pharmacokinetics following repeated dosing.1

Distribution

Extent

Does not cross blood-brain barrier.4

Not known whether taliglucerase alfa distributes into milk.1

Special Populations

Clearance in pediatric patients similar to that in adults.1

Elimination

Half-life

19–37 minutes.1

Stability

Storage

Parenteral

Powder for Injection

2–8°C. Protect from light.1

Following reconstitution, 2–8°C for up to 24 hours if protected from light or up to 4 hours at 20–25°C without protection from light.1

Following dilution, may store at 2–8°C for up to 24 hours; protect from light.1 Do not freeze.1

Compatibility

For information on systemic interactions resulting from concomitant use, see Interactions.

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).3 4 5 6 8 9

  • 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, and increased bone remineralization.1 3 6 7 8

Advice to Patients

  • Importance of informing patients that taliglucerase alfa is administered under the supervision of a clinician by IV infusion (over 1–2 hours) every other week.1

  • Risk of severe 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.

Taliglucerase Alfa

Routes

Dosage Forms

Strengths

Brand Names

Manufacturer

Parenteral

For injection, for IV infusion

200 units

Elelyso

Pfizer

AHFS DI Essentials. © Copyright 2017, Selected Revisions March 22, 2017. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.

References

1. Pfizer Labs. Elelyso (taliglucerase alfa) for injection, for intravenous use prescribing information. New York, NY; 2015 Nov.

2. Food and Drug Administration. FDA Application: Search Orphan Drug Designations and Approvals. Rockville, MD. From FDA website (http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm). Accessed 2012 Sep 14.

3. Cox TM. Gaucher disease: clinical profile and therapeutic developments. Biologics. 2010; 4:299-313. [PubMed 21209725]

4. Hollak CE. An evidence-based review of the potential benefits of taliglucerase alfa in the treatment of patients with Gaucher disease. Core Evid. 2012; 7:15-20. [PubMed 22654679]

5. Elstein D. Recent advances in treatment approaches to Gaucher disease. Curr Pharm Biotechnol. 2011; 12:854-60. [PubMed 21235447]

6. Genzyme Corporation. Cerezyme (imiglucerase) for injection prescribing information. Cambridge, MA; undated (accessed 2013 Jan 17).

7. Zimran A, Brill-Almon E, Chertkoff R et al. Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease. Blood. 2011; 118:5767-73. [PubMed 21900191]

8. US Food and Drug Administration. Center for Drug Evaluation and Research. Application number: 022458Orig1s000: Summary review. From FDA website. 2012 May 1.

9. Anon. In brief: Taliglucerase (Elelyso) for Gaucher disease. Med Lett Drugs Ther. 2012; 54:56.

11. Beutler E. Gaucher's disease. N Engl J Med. 1991; 325:1354-60. [PubMed 1922238]

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