Dosage Forms
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Solution Reconstituted, Intravenous [preservative free]:
Elelyso: 200 units (1 ea) [contains polysorbate 80]
Pharmacology
Mechanism of Action
Taliglucerase alfa is an analogue of glucocerebrosidase; it is produced by recombinant DNA technology using plant (carrot) cell culture. Glucocerebrosidase is an enzyme deficient in Gaucher's disease. It is needed to catalyze the hydrolysis of glucocerebroside to glucose and ceramide, thereby reducing liver and spleen size and improving anemia and thrombocytopenia.
Pharmacokinetics/Pharmacodynamics
Distribution
Distribution: Vss: Pediatric patients: 8.8 to 14.9 L; Adults: 10.7 to 11.7 L
Half-Life Elimination
Pediatric patients: 33 to 37 minutes; Adults: 19 to 29 minutes (dose-dependent; increased with higher doses)
Use: Labeled Indications
Gaucher disease: Treatment of adult and pediatric patients with a confirmed diagnosis of type 1 Gaucher disease.
Contraindications
There are no contraindications listed in the US labeling.
Canadian labeling: Severe hypersensitivity to taliglucerase alfa or any component of the formulation.
Dosage and Administration
Dosing: Adult
Note: Pretreatment with antihistamines and/or corticosteroids can be considered for prevention of subsequent infusion reactions in patients with an infusion reaction requiring symptomatic treatment; during clinical studies, patients were not routinely premedicated prior to infusion.
Gaucher disease (type 1): IV: 60 units/kg every 2 weeks; dosing is individualized based on disease severity.
Conversion from imiglucerase: Initiate taliglucerase alfa using the patient’s same previous imiglucerase dose and administer every 2 weeks. Note: Conversion to taliglucerase alfa is based on a single study of patients stabilized on a biweekly imiglucerase dose for ≥6 months
Dosing: Geriatric
Refer to adult dosing.
Dosing: Pediatric
Note: Pretreatment with antihistamines, antipyretics, and/or corticosteroids can be considered for prevention of subsequent infusion reactions in patients with an infusion reaction requiring symptomatic treatment; during clinical studies, patients were not routinely premedicated prior to infusion.
Gaucher disease (type 1): Children ≥4 years and Adolescents: IV: 60 units/kg/dose every 2 weeks
Conversion from imiglucerase: Initiate taliglucerase alfa using the patient’s same previous imiglucerase dose and administer every 2 weeks; doses may be adjusted if needed to achieve and maintain therapeutic goals. Note: Conversion to taliglucerase alfa is based on a single study of patients stabilized on a biweekly imiglucerase dose for ≥6 months.
Reconstitution
Calculate the necessary dose; round up to the next whole vial when determining the number of vials needed. Reconstitute each vial with 5.1 mL of SWFI; mix gently; do not shake. Solution should be clear and colorless. Withdraw 5 mL of reconstituted solution from each vial (reconstituted vials contain 5.3 mL) and further dilute in NS to a final volume of 100 to 200 mL. For pediatric patients, use a final volume of 100 to 120 mL; for adults, a final volume of 130 to 150 mL may be used. Final volume should not exceed 200 mL for adults using ≥130 to 150 mL or more of reconstituted product. Slight flocculation may occur following dilution; this is acceptable for administration. Discard any unused product.
Administration
IV: Administer IV over a minimum infusion time of 60 minutes (usual infusion time: 60 to 120 minutes). Administer using a low protein-binding infusion set with a 0.2 micron in-line filter.
Initiate infusion at a rate of 1.2 mL/minute; rate may be increased, but not exceed 2.2 mL/minute based on patient tolerance.
Storage
Store intact vials at 2°C to 8°C (36°F to 46°F); protect from light. Do not freeze. If not used immediately, the reconstituted solution may be stored at 2°C to 8°C (36°F to 46°F) for ≤24 hours (protect from light) or at 20°C to 25°C (68°F to 77°F) for ≤4 hours (without protection from light). The solution diluted in NS for infusion may be stored at 2°C to 8°C (36°F to 46°F) for ≤24 hours; protect from light. In total, the reconstituted and diluted products can be stored for ≤24 hours. Do not freeze.
Drug Interactions
There are no known significant interactions.
Adverse Reactions
>10%:
Central nervous system: Headache (13% to 19%)
Hypersensitivity: Hypersensitivity reaction (≤29%; increased risk in antibody-positive patients; patients switching from imiglucerase: 6%)
Immunologic: Antibody formation (≤53%; neutralizing 63%)
Neuromuscular & skeletal: Arthralgia (13%), limb pain (10%)
1% to <10%:
Cardiovascular: Flushing (6%)
Central nervous system: Dizziness (9%), fatigue (9%)
Dermatologic: Pruritus (6%), urticaria (6%)
Gastrointestinal: Nausea (9%), vomiting (≥6%), abdominal pain (6%)
Hypersensitivity: Anaphylaxis (3%)
Frequency not defined:
Hypersensitivity: Angioedema
Postmarketing: Back pain, diarrhea, fixed drug eruption (type III immune-mediated)
Warnings/Precautions
Concerns related to adverse effects:
- Antibody formation: The development of IgG anti-drug antibodies (ADA) has been reported; the clinical significance is unknown. Patients who develop immune or infusion reactions to taliglucerase alfa or who have had an immune response to other enzyme replacement therapies and who are switching to taliglucerase alfa should be monitored for antibody development; it is unknown if presence of antibodies is related to a higher risk of infusion reactions.
- CNS effects: Dizziness and fatigue have been observed with therapy; caution patients about performing dangerous tasks (eg, driving, operating machinery).
- Hypersensitivity/anaphylactoid reactions: Serious hypersensitivity reactions, including anaphylaxis, may occur; these reactions have occurred up to 3 hours after start of infusion. Appropriate medical support should be readily available. Base management of reaction on severity; may include slowing or temporary interruption of infusion and/or premedication (eg, antihistamine, antipyretics, corticosteroids) for mild reactions. Pretreatment may prevent subsequent reactions. Observe patient during and after infusion. If severe reactions occur, immediately discontinue infusion and initiate appropriate treatment; rechallenge with caution.
Monitoring Parameters
Hemoglobin, platelet count, angiotensin converting enzyme, tartrate-resistant acid phosphatase, chitotriosidase, IgG anti-drug antibody formation (in patients who experience, or previously experienced, immune or infusion reactions to enzyme replacement therapy); liver volume, spleen volume; bone density; ECG, echocardiogram, chest x-ray
Pregnancy
Pregnancy Considerations
Adverse effects were not observed in animal reproduction studies. 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 (which may result in increased bleeding and postpartum hemorrhage requiring transfusion), may occur.
Patient Education
What is this drug used for?
- It is used to treat Gaucher disease.
Frequently reported side effects of this drug
- Headache
- Joint pain
- Loss of strength and energy
- Abdominal pain
- Painful extremities
Other side effects of this drug: Talk with your doctor right away if you have any of these signs of:
- Severe dizziness
- Passing out
- Flushing
- Cough
- Nausea
- Vomiting
- Throat irritation
- Signs of a significant reaction like wheezing; chest tightness; fever; itching; bad cough; blue skin color; seizures; or swelling of face, lips, tongue, or throat.
Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.
Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a brief summary of general information about this medicine. It does NOT include all information about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not specific medical advice and does not replace information you receive from the healthcare provider. You must talk with the healthcare provider for complete information about the risks and benefits of using this medicine.
