Usual Adult Dose for Wolman's Disease

• Recommended dosage: 1 mg/kg as an IV infusion every other week
• Patients with suboptimal clinical response:
• Increase the dosage to 3 mg/kg IV every other week

• Suboptimal clinical response is defined as any of the following: poor growth, deteriorating biochemical markers such as alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or parameters of lipid metabolism such as low-density lipoprotein cholesterol (LDL-c), or triglycerides (TG).

Usual Pediatric Dose for Wolman's Disease

Infants with Rapidly Progressive LAL Deficiency within the first 6 months of life:

• Recommended dosage: 1 mg/kg IV once a week
• Patients with a suboptimal clinical response:
• Increase dosage to 3 mg/kg IV once a week
• Patients with continued suboptimal clinical response:
• Increase dosage to 5 mg/kg once a week

• Suboptimal clinical response is defined as any of the following: poor growth, deteriorating biochemical markers, or persistent or worsening organomegaly.

• Recommended dosage: 1 mg/kg as an IV infusion every other week
• Patients with suboptimal clinical response:
• Increase the dosage to 3 mg/kg IV every other week

• Suboptimal clinical response is defined as any of the following: poor growth, deteriorating biochemical markers such as alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or parameters of lipid metabolism such as low-density lipoprotein cholesterol (LDL-c), or triglycerides (TG).

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Precautions

US FDA requires a medication guide to assure safe use. For additional information: www.fda.gov/drugs/drug-safety-and-availability/medication-guides

CONTRAINDICATIONS: None

Safety and efficacy have not been established in patients younger than 1 month.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• For IV infusion only
• The solution should be clear. Inspect visually for particulate matter or discoloration prior to administration. Do not use if the solution is cloudy or particles are observed.
• Each vial is for single use. Discard any unused product.
• Consult the manufacturer product information for recommended infusion volumes.
• Infuse over at least 2 hours; consider a longer infusion time in patients receiving dosages greater than 1 mg/kg or in those who have had hypersensitivity reactions.
• An infusion rate of 1 hour may be considered in patients receiving the 1 mg/kg dose who tolerate the infusion

• Do not freeze or shake.
• Protect from light.
• Use diluted solution immediately; if cannot use immediately, may store for up to 24 hours at 2C to 8C (36F to 46F)

• The manufacturer product information should be consulted.

• Compatible with 0.9% Sodium Chloride for dilution

• Hypersensitivity: Signs/symptoms of hypersensitivity reaction (during and after infusion)

• Seek medical attention immediately if signs or symptoms of hypersensitivity or anaphylaxis occur.
• Read the Patient Information and Instructions for Use.

• Seek medical attention immediately if signs or symptoms of hypersensitivity or anaphylaxis occur.