Dosing & Uses
Dosage Forms & Strengths
- 2mg/mL (as 6mg/3mL single-use vial)
Dosage Forms & Strengths
- 2mg/mL (as 6mg/3mL single-use vial)
<5 years: Safety and efficacy not established
Shown to improve walking capacity in patients aged ≥5 years
In patients aged 16 months to 5 yr, no data are available to demonstrate improvement in disease-related symptoms or long term clinical outcome; however, treatment has reduced spleen volume similarly to that of adults and children aged 5 yr and older
Limb pain (28%)
Visual disturbance (22%)
Chest wall musculoskeletal pain (16%)
Musculoskeletal dysfunction (16%)
AE resulting from injury (13%)
Atrial abnormality (13%)
Infusion site edema (13%)
Pruritic rash (13%)
Skin disorder (13%)
Superficial injury (13%)
Frequency Not Defined
Loss of conaciousness
Black Box Warnings
Anaphylactoid reactions, which may be life threatening, have been observed in some patients during idursulfase infusions
Anaphylaxis, presenting as respiratory distress, hypoxia, hypotension, urticaria and/or angioedema of throat or tongue have been reported to occur during and after infusions, regardless of duration of the course of treatment
Appropriate medical support should be readily available when idursulfase is administered
Biphasic anaphylactic reactions have also been observed after idursulfase administration, and patients who have experienced anaphylactic reactions may require prolonged observation
Patients with compromised respiratory function or acute respiratory distress may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions and require additional monitoring
None listed in the manufacturer's label
Risk of anaphylactoid reactions; monitor patients with compromised respiratory function/acute respiratory disease
Patients with compromised respiratory function or acute febrile or respiratory illness at the time of infusion may be at higher risk of life-threatening complications from hypersensitivity reactions
Risk of hypersensitivity and antibody development is higher in individuals with seere genetic mutations (ie, complete gene deletion, large gene rearrangement, nonsense, frameshift or splice site mutations)
Caution when administering to patients susceptible to fluid overload, or patients with acute underlying respiratory illness or compromised cardiac and/or respiratory function for whom fluid restriction is indicated; risk of serious exacerbation of cardiac or respiratory status during infusions
Development of anti-idursulfase IgG antibodies reported in 51% of patients, which may increase incidence of infusion-related reactions
A registry has been created to track adverse effects, and monitor therapeutic responses during long-term treatment; may contact 1-866-888-0660 or www.eleprase.com
Pregnancy & Lactation
Pregnancy Category: C
Lactation: Unknown if distributed into human breast milk, use caution
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.
Mechanism of Action
Replaces iduronate-2-sulfatase enzyme, which hydrolyzes the mucopolysaccharides dermatan sulfate and heparan sulfate in various cells. Respiratory, cardiac and mobility dysfunction are caused by the accumulation of these polysacharides. Physical changes and CNS involvement also occur.
Peak plasma concentration: 1.1-1.5 mcg/mL
AUC: 169-206 min•mcg/mL
Vd: 21-25 L
Half-Life: 44-48 min
Clearance: 3-3.4 mL/min/kg
Do not infuse with other products in infusion tubing
Dilute calculated volume of drug in 100 mL 0.9% NaCl; mix gently
Discard if not administered/refrigerated within 8 hr
Diluted solution may be stored in refrigerator for 48 hr
Do not use if solution is discolored
Infuse IV over 1-3 hr; not to exceed 8 hr
Initial infusion rate: 8 mL/hr x15 min
If well tolerated, may increase by 8 mL/hr x15 min up to 100 mL/hr
Infusion may be slowed and/or temporarily stopped or discontinued
Refrigerate vials at 2-8°C (36-46°F); protect from light
Do not freeze/shake
Adding plans allows you to compare formulary status to other drugs in the same class.
To view formulary information first create a list of plans. Your list will be saved and can be edited at any time.
Adding plans allows you to:
- View the formulary and any restrictions for each plan.
- Manage and view all your plans together – even plans in different states.
- Compare formulary status to other drugs in the same class.
- Access your plan list on any device – mobile or desktop.
The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.
|1||This drug is available at the lowest co-pay. Most commonly, these are generic drugs.|
|2||This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.|
|3||This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.|
|4||This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.|
|5||This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.|
|6||This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.|
|NC||NOT COVERED – Drugs that are not covered by the plan.|
Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.