Dosing and uses of Elelyso (taliglucerase alfa)
Adult dosage forms and strengths
injection, lyophilized powder for reconstitution
- 200 units/vial
Gaucher Disease
Indicated for enzyme replacement in patients with confirmed diagnosis of type 1 (non-neuropathic) Gaucher disease
60 units/kg IV q2weeks initially
Administration
Reconstitute, dilute and administer under the supervision of a healthcare professionaL
Infuse IV over 1-2 hr
Adjust dose based on achievement and maintenance of each patient’s therapeutic goals; clinical trials have evaluated doses ranging from 11-73 units/kg every other week
Patients on a stable dose of imiglucerase can be switched to taliglucerase alfa at that same dose
Pediatric dosage forms and strengths
injection, lyophilized powder for reconstitution
- 200 units/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 q2weeks initially
Administration
Infuse IV over 1-2 hr
Adjust dose based on achievement and maintenance of each patient’s therapeutic goals; clinical trials have evaluated doses ranging from 11-73 units/kg every other week
Patients on a stable dose of imiglucerase can be switched to taliglucerase alfa at that same dose
Elelyso (taliglucerase alfa) adverse (side) effects
>10%
Vomiting, pediatrics (44%)
Hypersensitivity reactions (29%)
Headache (19%)
Arthralgia (13%)
1-10%
Fatigue (9%)
Nausea (9%)
Dizziness (9%)
Abdominal pain (6%)
Pruritus (6%)
Flushing (6%)
Vomiting (6%)
Urticaria (6%)
Serious hypersensitivity reactions, including anaphylaxis (2.8%)
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
Pregnancy and lactation
Pregnancy category: B
Lactation: Unknown whether distributed in breast milk; caution advised
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 of Elelyso (taliglucerase alfa)
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
Distribution
Vd: 7.3-11.7 L
Elimination
Half-life: 18.9-28.7 minutes
Systemic clearance: 20 L/hr (for 60 units/kg dose)
Administration
IV Preparation
Single use vial; do not use more than once
Remove required number of vials from refrigerator; do not leave at room temperature longer than 24 hr prior to reconstitution; do not heat or microwave
Using aseptic technique, reconstitute the vial with 5.1 mL sterile water for injection to yield 5.3 mL and a withdrawal volume of 5 mL
Upon reconstitution, mix vials gently (DO NOT SHAKE)
Prior to further dilution, visually inspect the solution in the vials; the solution should be clear and colorless; do not use if discolored or foreign particulate matter is present
Withdraw 5 mL (ie, 200 units/5 mL) of reconstituted solution from each vial and dilute with 0.9% NaCl to a final volume of 100-120 mL for pediatric patients or 130-15 mL for adults
Use a low-protein-binding container
IV Administration
Administered with a low-protein-binding infusion set equipped with an in-line, low-protein-binding 0.2 micrometer filter
Adults: Initial IV infusion rate of 1.2 mL/minute; may increase to 2.2 mL/minute after patient tolerability established
Pediatrics: Initial IV infusion rate of 1mL/minute; may increase to 2 mL/minute after patient tolerability established
The total volume of the infusion solution should be delivered over a period of at least 1 hour
Storage
Unopened vials
- Refrigerate unopened vials between 2-8°C (36-46°F)
- Protect vials from light
Reconstituted & diluted solution
- Contains no preservative, the product should be used immediately once reconstituted; if immediate use is not possible, the reconstituted or the diluted product may be stored for up to 24 hours at 2-8°C (36-46°F)
- Do not freeze
- Protect from light
- Discard any unused product
