Usual Adult Dose for Sphingolipidosis

Recommended starting dose: 0.1 mg/kg IV infusion

Dose Escalation Regimen:
First dose (Day 1/Week 0): 0.1 mg/kg IV as a single dose
Second dose (Week 2): 0.3 mg/kg IV as a single dose
Third dose (Week 4): 0.3 mg/kg IV as a single dose
Fourth dose (Week 6): 0.6 mg/kg IV as a single dose
Fifth dose (Week 8): 0.6 mg/kg IV as a single dose
Sixth dose (Week 10): 1 mg/kg IV as a single dose
Seventh dose (Week 12): 2 mg/kg IV as a single dose
Eighth dose (Week 14): 3 mg/kg IV as a single dose

Maintenance dose: 3.0 mg/kg IV infusion every 2 weeks

Comments:

• Individualize the dose based on the patient's weight.
• For body mass index (BMI) less than 30, use actual body weight (kg).
• For BMI greater than 30, use adjusted body weight (kg).
• Adjusted body weight can be calculated by squaring the patient's actual height in meters and then multiplying by 30. The manufacturer product information should be consulted.
• Obtain baseline transaminase levels within 1 month prior to treatment initiation in all patients.
• Verify the pregnancy status of females of reproductive potential.
• Prior to administration, consider pretreating with antihistamines, antipyretics, or corticosteroids.
• Do not infuse this drug into the same intravenous line as other products.

Use: For the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients

Usual Pediatric Dose for Sphingolipidosis

Less than 18 years:
Recommended starting dose: 0.03 mg/kg IV infusion

Dose Escalation Regimen:

• First dose (day 1/week 0): 0.03 mg/kg IV as a single dose
• Second dose (week 2): 0.1 mg/kg IV as a single dose
• Third dose (week 4): 0.3 mg/kg IV as a single dose
• Fourth dose (week 6): 0.3 mg/kg IV as a single dose
• Fifth dose (week 8): 0.6 mg/kg IV as a single dose
• Sixth dose (week 10): 0.6 mg/kg IV as a single dose
• Seventh dose (week 12): 1 mg/kg IV as a single dose
• Eighth dose (week 14): 2 mg/kg IV as a single dose
• Ninth dose (week 16): 3 mg/kg IV as a single dose

Maintenance dose: 3.0 mg/kg IV infusion every 2 weeks

Comments:

• Individualize the dose based on the patient's weight.
• For body mass index (BMI) less than 30, use actual body weight (kg).
• For BMI greater than 30, use adjusted body weight (kg).
• Adjusted body weight can be calculated by squaring the patient's actual height in meters and then multiplying by 30. The manufacturer product information should be consulted.
• Obtain baseline transaminase levels within 1 month prior to treatment initiation in all patients.
• Verify pregnancy status in females of reproductive potential.
• Prior to administration, consider pretreating with antihistamines, antipyretics, or corticosteroids.
• Do not infuse this drug into the same intravenous line with other products.

Use: For the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Dose Adjustments

• If severe hypersensitivity or infusion-associated reactions occur, then immediately discontinue dose administration, and initiate appropriate medical treatment.
• Temporarily interrupt or slow down the infusion rate in the case of mild to moderate hypersensitivity or infusion-associated reactions. If the dose is reduced, re-escalate following Dose Escalation Regimen guidelines.
• In the case of elevated transaminase levels, adjust the dose or temporarily withhold therapy until the liver transaminase levels return to baseline.

Precautions

US BOXED WARNING:

SEVERE HYPERSENSITIVITY REACTIONS:

• Hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with this drug.

Recommendation:

• Appropriate medical support measures and equipment should be readily available during administration.
• If a severe hypersensitivity reaction occurs, discontinue use of this drug, and initiate appropriate medical treatment.
• In patients with a severe hypersensitivity reaction a desensitization procedure may be instituted.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• Before administration, inspect the syringe or infusion bag for foaming. If foaming is present, let the foam dissipate.
• Consider pretreating with antihistamines, antipyretics, and/or corticosteroids.
• Use an in-line low protein-binding 0.2-micron filter during administration.
• Infuse the drug using the infusion rates described in the manufacturer's product information.

Storage requirements:
Unreconstituted Vials:

• Refrigerated at 2C to 8C (36F to 46F).
• Do not freeze

Reconstituted and Diluted Solution:

• If the reconstituted vial or diluted solution is not used or administered immediately, store refrigerated (2C to 8C [36F to 46F]) for up to 24 hours or at room temperature (20C to 25C [68F to 77F]) for up to 12 hours.
• Do not freeze

Preparation techniques: The manufacturer product information should be consulted.

General:

• The carcinogenic and mutagenic potential of this drug has not been studied.

Patient advice:

• In the case of missed doses or delayed infusion, contact the physician.

Frequently asked questions

• How effective is Xenpozyme?