elosulfase alfa (Rx)

Brand and Other Names:Vimizim
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Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

concentrated intravenous solution

  • 1mg/mL (in 5mg/5mL single use vials [requires dilution])

Morquio A Syndrome

Indicated for enzyme replacement therapy for Morquio A syndrome (mucopolysaccharidosis Type IVA [MPS IVA])

2 mg/kg IV infused over a minimum of 3.5-4.5 hr

Dosing Considerations

Inform patients of the Morquio A Registry established in order to better understand the variability and progression of the disease in the population as a whole, and to monitor and evaluate long-term treatment effects of elosulfase alfa

For more information, contact MARS@bmrn.com or call 1-800-983-4587

Administration

Pretreat with antihistamines (with or without antipyretics) 30-60 minutes before initiating infusion

Dosage Forms & Strengths

concentrated intravenous solution

  • 1mg/mL (in 5mg/5mL single use vials [requires dilution])

Morquio A Syndrome

Enzyme replacement therapy pending FDA approval for Morquio A syndrome (mucopolysaccharidosis Type IVA [MPS IVA])

<5 years: Safety and efficacy not established

≥5 years: 2 mg/kg IV infused over a minimum of 3.5-4.5 hr

Dosing Considerations

Inform patients of the Morquio A Registry established in order to better understand the variability and progression of the disease in the population as a whole, and to monitor and evaluate long-term treatment effects of elosulfase alfa

For more information, contact MARS@bmrn.com or call 1-800-983-4587

Administration

Pretreat with antihistamines (with or without antipyretics) 30-60 minutes before initiating infusion

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Adverse Effects

>10%

Pyrexia (33%)

Vomiting (31%)

Headache (26%)

Nausea (24%)

Abdominal pain (21%)

Hypersensitivity, including anaphylaxis (18.7%)

Chills (10.3%)

Fatigue (10.3%)

1-10%

Anaphylaxis (7.7%)

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Warnings

Black Box Warnings

Anaphylaxis

  • Life-threatening anaphylactic reactions reported during IV infusion
  • Anaphylaxis, presenting as cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, and gastrointestinal symptoms (eg, nausea, abdominal pain, retching, and vomiting) in conjunction with urticaria, have been reported to occur during infusions, regardless of duration of the course of treatment
  • Closely observe patients during and after administration and be prepared to manage anaphylaxis
  • Inform patients of the signs and symptoms of anaphylaxis and have them seek immediate medical care should symptoms occur Patients with acute respiratory illness may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions, and require additional monitoring

Contraindications

None

Cautions

Anaphylaxis reported and may occur as early as 30 minutes from the start of infusion and up to 3 hr after completing the infusion (see Black Box Warnings)

Increased risk of acute respiratory complications; monitor patients with acute febrile or respiratory illness at the time of infusion (higher risk of life-threatening complications from hypersensitivity reaction)

Sleep apnea is common in patients with MPS IVA, evaluate airway patency before initiating treatment with elosulfase alfa

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Pregnancy & Lactation

Pregnancy Category: C

Lactation: Unknown if distributed in human breast milk, use with caution; present in milk from treated rats

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.

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Pharmacology

Mechanism of Action

Replaces deficient enzyme N-acetylgalactosamine-6 sulfatase (GALNS) to minimize progressive multisystemic manifestations of Morquio A syndrome

Sulfatase activity deficiency results in the accumulation of the GAG substrates, KS and C6S, in the lysosomal compartment of cells throughout the body; the accumulation leads to widespread cellular, tissue, and organ dysfunction

Enzyme replacement provides the exogenous enzyme GALNS that will be taken up into the lysosomes and increase catabolism of GAGs KS and C6S

Absorption

All measurements at treatment week 22

AUC: 577 min•mcg/mL

Peak plasma time: 202 minutes

Peak plasma concentration: 4.04 mcg/mL

Distribution

All measurements at treatment week 22

Vd: 7.08 mL/min/kg

Elimination

All measurements at treatment week 22

Half-life: 35.9 minutes

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Administration

IV Compatibilities

Do not infuse with other products in the infusion tubing

Compatibility with other products has not been evaluated

IV Preparation

Determine the number of vials to be diluted based on the individual patient’s weight and the recommended dose of 2 mg/kg

The solution should be clear to slightly opalescent and colorless to pale yellow when diluted

Do not use if the solution is discolored or if there is particulate matter in the solution

Note that a diluted solution with slight flocculation (eg, thin translucent fibers) is acceptable for administration

Avoid agitation during preparation; gently rotate the bag to ensure proper distribution

Do NOT shake the solution

Dilute the calculated dose to a final volume of 100 mL or 250 mL using 0.9% NaCl

Final volume is based on the patient’s weight

  • <25 kg: 100 mL
  • ≥25 kg: 250 mL

IV Administration

Administer diluted solution IV using a low-protein binding infusion set equipped with a low-protein binding 0.2 micrometer in-line filter

The infusion rate may be slowed, temporarily stopped, or discontinued for that visit in the event of hypersensitivity reactions

Weight <25 kg

  • Diluted volume totals 100 mL
  • 3 mL/hr IV initially for the first 15 minutes, and if tolerated, increased to 6 mL/hr for the next 15 minutes
  • If this rate is tolerated, then the rate may be increased every 15 minutes in 6 mL/hr increments, not to exceed 36 mL/hr
  • The total volume of the infusion should be delivered over a minimum of 3.5 hr

Weight ≥25 kg

  • Diluted volume totals 250 mL
  • 6 mL/hr IV initially for the first 15 minutes, and if tolerated, increased to 12 mL/hr for the next 15 minutes
  • If this rate is tolerated, then the rate may be increased every 15 minutes in 12 mL/hr increments, not to exceed 72 mL/hr
  • The total volume of the infusion should be delivered over a minimum of 4.5 hr

Storage

Contains no preservatives; discard partially used vials

Do not freeze

Do not shake

Protect from light

Unopened vials

  • Store refrigerated between 2-8°C [36-46°F]) in original carton and protect from light

Diluted solution

  • If immediate use is not possible, store refrigerated for up to 24 hr at 2-8°C (36-46°F) followed by up to 24 hr at 23-27°C (73-81°F) during administration
  • Administration should be completed within 48 hr from the time of dilution
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Formulary

FormularyPatient Discounts

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.
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  • Compare formulary status to other drugs in the same class.
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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.

Tier Description
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.
Code Definition
PA Prior Authorization
Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
QL Quantity Limits
Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
ST Step Therapy
Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
OR Other Restrictions
Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.