Usual Adult Dose for Mucopolysaccharidosis Type IV

2 mg/kg via IV infusion once a week

Comments:

• Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes before starting the infusion.
• See Other Comments/Administration Advice for recommended dilution volume and infusion rates.

Usual Pediatric Dose for Mucopolysaccharidosis Type IV

5 years or older: 2 mg/kg via IV infusion once a week

Comments:

• Pretreatment with antihistamines with or without antipyretics is recommended 30 to 60 minutes before starting the infusion.
• See Other Comments/Administration Advice for recommended dilution volume and infusion rates.

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Precautions

US BOXED WARNING: RISK OF ANAPHYLAXIS

• Life-threatening anaphylactic reactions reported in some patients during infusions of this drug. Anaphylaxis occurred during infusions, regardless of treatment duration. Patients should be observed closely during and after infusion and healthcare providers should be prepared to manage anaphylaxis. Patients should be advised of the signs/symptoms of anaphylaxis and should seek medical care at once if 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.

Dialysis

Data not available

Other Comments

Administration advice:
For IV infusion: Administer diluted solution using a low-protein-binding infusion set with a low-protein-binding 0.2 micrometer in-line filter

• Patients weighing less than 25 kg: Infusion volume should be 100 mL delivered over a minimum of 3.5 hours; initial infusion rate should be 3 mL/hr for the first 15 minutes, if tolerated, increase to 6 mL/hr for next 15 minutes; then, continue to increase in 6 mL/hr increments as tolerated not to exceed 36 mL/hr
• Patients weighing 25 kg or more: Infusion volume should be 200 mL delivered over a minimum of 4.5 hours; initial infusion rate should be 6 mL/hr for the first 15 minutes, if tolerated, increase to 12 mL/hr for next 15 minutes; then, continue to increase in 12 mL/hr increments as tolerated not to exceed 72 mL/hr
• Infusion rates may be slowed, temporarily stopped, or discontinued in the event of hypersensitivity reactions

• Dilute to a final volume of 100 mL (weight less than 25 kg) or 250 mL (weight 25 kg or more) using 0.9% sodium chloride injection
• Avoid agitation during preparation; gently rotate bag to ensure proper distribution
• Complete infusion within 48 hours from the time of dilution

• Do not freeze or shake
• Protect from light
• Concentrated solution: Store at 2C to 8C (36F to 46F)
• Diluted solution: If cannot use immediately, may store for up to 24 hours at 2C to 8C (36F to 46F) followed by up to 24 hours at 23C to 27C (73F to 81F)

• Compatible with 0.9% Sodium Chloride Injection, USP
• Should not be infused with other products in the infusion tubing; compatibility not evaluated

• Closely observe patients during and after administration; infusions should occur in a setting equipped to treat anaphylaxis.
• Clinical studies did not include patients 65 years or older so it is unknown whether they respond differently.

• Patients/caregivers should understand that anaphylactic and hypersensitivity reactions may occur beyond the infusion time; in the event of a severe reaction, seek immediate medical care.
• A voluntary Morquio A Registry has been established. For additional information and enrollment: [email protected]