Dosing & Uses
Dosage Forms & Strengths
injection, IV solution
- 2mg/mL (10mg/5mL single-dose vial)
Mucopolysaccharidosis VII
Indicated for mucopolysaccharidosis VII (MPS VII, Sly syndrome) in adult and pediatric patients
Premedication 30-60 minutes before IV infusion with a nonsedating antihistamine with or without an antipyretic medication is recommended
See also Administration for precise infusion rate instructions
Dosing Considerations
Observe patients closely during the infusion and following the infusion for a minimum of 60 minutes for the development of anaphylaxis
Limitation of use
- The effect on CNS manifestations of MPS VII has not been determined
Dosage Forms & Strengths
injection, IV solution
- 2mg/mL (10mg/5mL single-dose vial)
Mucopolysaccharidosis VII
Indicated for mucopolysaccharidosis VII (MPS VII, Sly syndrome) in adult and pediatric patients
Premedicate 30-60 minutes before IV infusion with a nonsedating antihistamine with or without an antipyretic medication is recommended
See also Administration for precise infusion rate instructions
Dosing Considerations
Observe patients closely during the infusion and following the infusion for a minimum of 60 minutes for the development of anaphylaxis
Limitation of use
- The effect on CNS manifestations of MPS VII has not been determined
Interactions
Interaction Checker
No Results

Contraindicated
Serious - Use Alternative
Significant - Monitor Closely
Minor

Contraindicated (0)
Serious - Use Alternative (0)
Monitor Closely (0)
Minor (0)
Adverse Effects
<1%
Extravasation (0.5%)
Diarrhea (0.4%)
Rash (0.4%)
Anaphylaxis (0.4%)
Infusion site swelling (0.1%)
Peripheral swelling (0.1%)
Pruritus (0.1%)
Warnings
Black Box Warnings
Anaphylaxis has occurred as early as the first dose; therefore, appropriate medical support should be readily available during IV infusion
Closely observe patient during and for 60 minutes after infusion
Immediately discontinue treatment if anaphylaxis occurs
Contraindications
None
Cautions
Anaphylaxis was reported in the clinical program; reactions occurred during infusion and were observed as early as the first dose; manifestations included respiratory distress, cyanosis, decreased oxygen saturation, and hypotension; administer under supervision of a healthcare professional with experience managing anaphylaxis (see Dosing Considerations and Black Box Warnings)
Pregnancy & Lactation
Pregnancy
No data are available regarding use in pregnant women
When administered to pregnant rats and rabbits during organogenesis, vestronidase alfa showed no maternal toxicity or adverse developmental outcomes at doses causing maternal serum exposures (AUC) up to 1.6 and 10 times, respectively for rats and rabbits, the exposure at the recommended human dose
Lactation
Unknown if present in either human or animal milk; the effects on the breastfed infant, or the 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
Mucopolysaccharidosis VII (MPS VII or Sly syndrome) is a lysosomal disorder characterized by the deficiency of beta glucuronidase (GUS) that results in GAG accumulation in cells throughout the body, leading to multisystem tissue and organ damage
Recombinant human lysosomal GUS is intended to provide exogenous GUS enzyme for uptake into cellular lysosome; mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to cell surface receptors, leading to cellular uptake of the enzyme, targeting to lysosomes and subsequent catabolism of accumulated GAGs in affected tissues
Absorption
Peak plasma concentration: 20 mcg/mL (range: 6.6-34.9 mcg/mL)
AUC: 3440 mcg·min/mL (range: 1130- 5820 mcg·min/mL)
Distribution
Vd: 260 mL/kg (range: 97-598 mL/kg)
Metabolism
Vestronidase alfa is a recombinant human enzyme and is therefore eliminated by proteolytic degradation into small peptides and amino acids
Elimination
Clearance: 1.3 mL/min/kg (range: 0.6 to 3.3 mL/min/kg)
Half-life: 155 minutes (range: 51-213 minutes)
No excretion studies have been conducted in humans; not expected to be eliminated through urine or feces
Administration
IV Compatibility
0.9% NaCl
Compatibility with other products has not been evaluated
IV Preparation
Determine total dose based on patient’s actual body weight (refer to prescribing information)
Remove required number of vials from refrigerator and allow them to reach room temperature; do not heat, microwave, or shake vials
Visually inspect solution for particulate matter and discoloration; slight flocculation (thin translucent fibers) may appear; solution should appear colorless to slightly yellow
Slowly withdraw the calculated dose using a large needle (eg, 18 gauge) to minimize bubbles in the solution
Add 0.9% NaCl equal to the drug volume to infusion bag; final solution should be a 1:1 solution of vestronidase alfa to 0.9% NaCl
Diluted solutions may be refrigerated (2-8°C [36-46°F]) for up to 36 hr and may be stored at room temperature (25°C [77°F]) for up to 6 hr
IV administration
Use an infusion set with an inline, low-protein binding 0.2 micron filter
Do not flush the line containing vestronidase alfa to avoid a rapid bolus of infused enzyme
Owing to the low infusion rate, additional saline may be added through a separate line (piggyback or Y tube) to maintain sufficient intravenous flow to prevent clotting or line blockage
Do not infuse with other products in the infusion tubing
Use immediately after dilution and complete the infusion within 42 hr from the time of dilution
Discard any unused product
Rate of infusion
- First hour: Infuse 2.5% of the total volume; account for any dead space in the lines to ensure 2.5% of the total infusion volume is delivered
- Subsequent 3 hours: Infuse remaining volume (refer to prescribing information)
Storage
Unused vials: Store under refrigeration at 2-8°C (36-46°F)
Diluted solutions: Store in refrigerator at 2-8°C (36-46°F) for up to 36 hr and may be stored at room temperature at 25°C (77°F) for up to 6 hr
Do no freeze or shake
Protect from light
Images
Formulary
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.