Usual Adult Dose for Mucopolysaccharidosis Type VII

4 mg/kg IV infusion once every 2 weeks

Use: For the treatment of Mucopolysaccharidosis VII (MPS VII) also known as Sly syndrome

Usual Pediatric Dose for Mucopolysaccharidosis Type VII

4 mg/kg IV infusion once every 2 weeks

Use: For the treatment of Mucopolysaccharidosis VII (MPS VII) also known as Sly syndrome

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Precautions

US BOXED WARNING:

• ANAPHYLAXIS: Anaphylaxis has occurred with administration of this drug, as early as the first dose, therefore, appropriate medical support should be readily available when this drug is administered. Closely observe patients during and for 60 minutes after infusion. Immediately discontinue the infusion if the patient experiences anaphylaxis.

Dialysis

Data not available

Other Comments

Administration advice:

• For IV infusion only.
• Administer the diluted solution using a low protein binding infusion set with an in-line, low protein binding 0.2 micron filter.
• This drug should be administered under the supervision of a healthcare professional with the ability to manage anaphylaxis.
• Premedication is recommended 30 to 60 minutes prior to the start of the infusion, including a nonsedating antihistamine with or without an antipyretic.
• Administer infusion over about 4 hours; infuse the first 2.5% of the total volume over the first hour; after the first hour, increase the infusion rate as tolerated in order to complete infusion over the following 3 hours according to the recommended rate guidelines.
• Observe patients closely during infusion and for at least 60 minutes after the infusion for the development of anaphylaxis.
• Discontinue infusion immediately if severe systemic reaction, including anaphylaxis, occurs.
• The manufacturer product information should be consulted for further information, including rate of infusion.

• Store vials at 2C to 8C (36F to 46F); do not freeze or shake; protect from light.
• Diluted solution: If not used immediately, diluted solution may be stored up to 36 hours at 2C to 8C (36F to 46F) followed by up to 6 hours at room temperature up to a maximum of 25C (77F).
• Any unused product should be discarded.

• The manufacturer product information should be consulted.

• Do not infuse with other products in the infusion tubing.
• Do not flush the line containing this drug to avoid a rapid bolus of infused enzyme; due 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.

• The effect of this drug on the central nervous system manifestations of MPS VII has not been established.

• Immunologic: Observe patients during and for at least 60 minutes after infusion for severe reactions, including anaphylaxis.

• Seek immediate medical care if signs/symptoms of anaphylaxis occur.