Dosing & Uses
Dosage Forms & Strengths
injection, lyophilized powder for reconstitution
- 200units/vial
Gaucher Disease
Indicated for treatment of Type 1 Gaucher disease in patients aged ≥4 years with a confirmed diagnosis
Treatment-naïve
- 60 units/kg IV every other week
Switching from imiglucerase
- Currently on stable imiglucerase dose: Begin taliglucerase alfa at same units/kg dosage of imiglucerase when switching from imiglucerase to taliglucerase alfa
- Administer for long-term treatment as IV infusion every other week
- Adjust dosage based on achievement and maintenance each patient’s therapeutic goals
Dosage Forms & Strengths
injection, lyophilized powder for reconstitution
- 200units/vial
Gaucher Disease
Indicated for enzyme replacement in patients with confirmed diagnosis of type 1 (non-neuropathic) Gaucher disease
<4 years: Safety and efficacy not established
≥4 years: 60 units/kg IV every other week
Switching from imiglucerase
- Currently on stable imiglucerase dose: Begin taliglucerase alfa at same units/kg dosage of imiglucerase when switching from imiglucerase to taliglucerase alfa
- Administer for long-term treatment as IV infusion every other week
- Adjust dosage based on achievement and maintenance each patient’s therapeutic goals
Adverse Effects
>10%
Treatment-naïve adults
- Headache (19%)
- Arthralgia (13%)
Treatment naïve children and adolescents
- Vomiting (≥10%)
Switch from imiglucerase adults and children
- Arthralgia (13%)
- Headache (13%)
1-10%
Treatment-naïve adults
- Fatigue (9%)
- Nausea (9%)
- Dizziness (9%)
- Abdominal pain (6%)
- Pruritus (6%)
- Flushing (6%)
- Vomiting (6%)
- Urticaria (6%)
Switch from imiglucerase adults and children
- Pain in extremity (10%)
Postmarketing Reports
Gastrointestinal disorders: Vomiting, diarrhea
General disorders and administration site conditions: Fatigue
Immune system disorders: Anaphylaxis, Type III immune-mediated fixed drug eruption
Musculoskeletal and connective tissue disorders: Back pain
Warnings
Contraindications
None
Cautions
Anaphylaxis reported; if anaphylaxis occurs, discontinue infusion immediately and initiate appropriate treatment
Allergic and infusion reactions are common and include headache, chest pain or discomfort, asthenia, fatigue, urticaria, erythema, increased blood pressure, back pain, arthralgia, and flushing; if allergic or infusion reactions occur, decrease the infusion rate or temporarily stop the infusion; antihistamines and/or antipyretics may be administered; medical support should be readily available when taliglucerase alfa administered; observe patients closely for an appropriate period of time after administration; reactions have occurred up to 3 hr after the start of infusion
Consider risks and benefits of re-administering taliglucerase alfa in patients who have experienced severe reaction associated with taliglucerase alfa; use caution upon rechallenge; appropriate medical support should be readily available
As with all therapeutic proteins, development of IgG anti-drug antibodies (ADA) has been reported (53%); unknown if presence of ADA is associated with higher risk of infusion reactions or immunogenicity
Monitor patients that have had an immune response to other enzyme replacement therapies or who are switching to taliglucerase alfa; unknown if these patients are at higher risk of infusion reactions
Dizziness and fatigue reported; caution patients against performing dangerous tasks that require mental alertness, including driving heavy machinery
Hypersensitivity reactions
- Reactions, including serious hypersensitivity reactions such as anaphylaxis, reported; due to potential for anaphylaxis, appropriate medical support should be readily available during infusion
- Observe patients closely for 3 hr after start of infusion; inform patients of signs and symptoms of anaphylaxis, and instruct them to seek immediate medical care should signs and symptoms occur
- Base management of hypersensitivity reactions on severity of reaction and should include slowing or temporary interruption of infusion and/or administration of antihistamines, antipyretics, and/or corticosteroids for mild reactions
- To reduce risk of hypersensitivity reactions, consider pretreatment with antihistamines and/or corticosteroids; if severe hypersensitivity reactions including anaphylaxis occur, immediately stop infusion and initiate appropriate treatment
- Consider risks and benefits of re-administering therapy in patients who have experienced severe hypersensitivity reaction associated with this medication; exercise caution upon rechallenge
Pregnancy & Lactation
Pregnancy
Insufficient data available on use in pregnant females to inform a drug-associated risk
Animal data
- When pregnant rats and rabbits were administered IV taliglucerase alfa at doses up to 5 times the recommended human dose, there was no evidence of embryofetal toxicity
Clinical considerations
- Women with Type 1 Gaucher disease have an increased risk of spontaneous abortion if disease symptoms are not treated and controlled preconception and during pregnancy
- Pregnancy may exacerbate existing Type 1 Gaucher disease symptoms or result in new disease manifestations
- Type 1 Gaucher disease manifestations may lead to adverse pregnancy outcomes, including hepatosplenomegaly which can interfere with the normal growth of a fetus and thrombocytopenia which can lead to increased bleeding and possible postpartum hemorrhage requiring transfusion
Lactation
There are no data on taliglucerase alfa in human milk, effects on breast fed infants, or effects on milk production
Pregnancy Categories
A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.
B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk. C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done. D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk. X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist. NA: Information not available.Pharmacology
Mechanism of Action
Gaucher disease is caused by a deficiency of the enzyme to catalyze glucocerebrosidase resulting in lipid accumulation in the spleen, liver, kidneys, and other organs; major signs include liver or spleen damage, anemia, and thrombocytopenia
Taliglucerase alfa catalyzes the hydrolysis of glucocerebroside to glucose and ceramide which results in reduced spleen and liver enlargement, and increased RBCs and platelets
Absorption
AUC
- Children and adolescents (30 units/kg): 1416 ng⋅hr/mL
- Children and adolescents (60 units/kg): 2984 ng⋅hr/mL
- Adults (30 units/kg): 2007 ng⋅hr/mL
- Adults (60 units/kg): 6459 ng⋅hr/mL
Distribution
Vd (steady-state)
- Children and adolescents (30 units/kg): 14.9 L
- Children and adolescents (60 units/kg): 8.8 L
- Adults (30 units/kg): 11.7 L
- Adults (60 units/kg): 10.7 L
Elimination
Clearance
- Children and adolescents (30 units/kg): 30.5 L/hr
- Children and adolescents (60 units/kg): 15.8 L/hr
- Adults (30 units/kg): 30.5 L/hr
- Adults (60 units/kg): 18.5 L/hr
Half-life
- Children and adolescents (30 units/kg): 37.1 min
- Children and adolescents (60 units/kg): 32.5 min
- Adults (30 units/kg): 18.9 min
- Adults (60 units/kg): 28.7 min
Administration
IV Preparation
Remove required number of vials from refrigerator; do not leave vials at room temperature >24 hr before reconstitution; do not heat or microwave vials
Reconstitute each vial with 5.1 mL of Sterile Water for Injection to yield concentration of 40 units/mL
Upon reconstitution, mix vials gently; DO NOT SHAKE
Before further dilution, visually inspect vials; solution should be clear and colorless; discard if solution is discolored or if foreign particulate matter is present
Withdraw calculated dose from reconstituted vials and dilute with 0.9% NaCl, to a final volume of 100-120 mL (in children and adolescents) and 130-150 mL (in adults) should be used
For adults, if volume of reconstituted product alone ≥130 to 150 mL, then final volume should not exceed 200 mL
Mix gently; DO NOT SHAKE
Since this is a protein solution, slight flocculation (described as translucent fibers) occurs occasionally after dilution
IV Administration
Infuse diluted solution IV and filtered through in-line, low protein-binding 0.2 micron filter
Pediatric patients weighing <30 kg (actual body weight): 1 mL/min
Pediatric patients weighing ≥30 kg (actual body weight): 1 mL/min initially; may increase up to 2 mL/min after tolerability established
Adults: 1.2 mL/min initially; may increase up to 2.2 mL/min after tolerability established
Storage
Unused vials
- Refrigerate at 2-8ºC (36-46ºF); protect from light
Reconstituted vials
- Contains no preservative
- Use immediately once reconstituted
- If immediate use not possible, refrigerate at 2-8ºC (36-46ºF) for up to 24 hr; protect from light OR
- Store at room temperature (20-25ºC [68-77ºF]) for up to 4 hr without protection from light
Diluted solution
- Refrigerate at 2-8ºC (36-46ºF) for up to 24 hr; protect from light
- Do not freeze; discard any unused product
Images
BRAND | FORM. | UNIT PRICE | PILL IMAGE |
---|---|---|---|
Elelyso intravenous - | 200 unit vial | ![]() |
Copyright © 2010 First DataBank, Inc.
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