Usual Adult Dose for Pompe disease

Less than 30 kg: 40 mg/kg IV every 2 weeks
At least 30 kg: 20 mg/kg every 2 weeks

Comments:

• The patient's actual body weight should be used to calculate the dose.

Usual Pediatric Dose for Pompe disease

1 year or older:

• Less than 30 kg: 40 mg/kg IV every 2 weeks
• At least 30 kg: 20 mg/kg every 2 weeks

• The patient's actual body weight should be used to calculate the dose.

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Dose Adjustments

Severe hypersensitivity reaction (including anaphylaxis) or severe infusion-associated reaction (IAR):

• Administration of this drug should be immediately discontinued and appropriate medical treatment should be started.

• Temporarily holding or slowing the infusion rate should be considered and appropriate medical treatment should be started.
• If symptoms persist despite temporarily holding the infusion: The treating physician should wait at least 30 minutes for symptoms to resolve before deciding to stop the infusion for the day.
• If symptoms subside: The infusion should resume for 30 minutes at half the rate at which the reaction occurred, and then the infusion rate should be increased by 50% for 15 to 30 minutes; if symptoms do not recur, the infusion rate should be increased to the rate at which the reaction occurred and subsequent increases in a stepwise manner should be considered.

Precautions

US BOXED WARNINGS:

• HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Patients treated with this drug have had life-threatening hypersensitivity reactions (including anaphylaxis); appropriate medical support measures (including cardiopulmonary resuscitation equipment) should be readily available during administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, this drug should be discontinued immediately and appropriate medical treatment should be started; a desensitization procedure to this drug may be considered in patients with severe hypersensitivity reaction.
• INFUSION-ASSOCIATED REACTIONS (IARs): Patients treated with this drug have had severe IARs; if severe IARs occur, immediate discontinuation of this drug, starting appropriate medical treatment, and the benefits/risks of readministering this drug after severe IARs should be considered. Patients with an acute underlying illness at the time of infusion may be at greater risk for IARs; patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to higher risk of severe complications from IARs.
• RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function for whom fluid restriction is indicated may be at risk of serious exacerbation of their cardiac or respiratory status during infusion of this drug. More frequent monitoring of vitals should be performed during infusion in such patients.

Dialysis

Data not available

Other Comments

Administration advice:

• Before administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids.
• Administer as an IV infusion; use an in-line, low protein binding, 0.2 micrometer filter.
• The initial infusion rate is 1 mg/kg/hour; gradually increase the infusion rate every 30 minutes if there are no signs of IARs.
• For 20 mg/kg (initial and subsequent infusions): After the initial infusion rate, increase in 3 steps: 3 mg/kg/hour, 5 mg/kg/hour, and then 7 mg/kg/hour; then, maintain the infusion rate at 7 mg/kg/hour until the infusion is complete. Approximate total infusion duration: 4 to 5 hours
• For 40 mg/kg (initial infusion): After the initial infusion rate, increase in 3 steps: 3 mg/kg/hour, 5 mg/kg/hour, and then 7 mg/kg/hour; then, maintain the infusion rate at 7 mg/kg/hour until the infusion is complete (4-step process). Approximate total infusion duration: 7 hours
• For 40 mg/kg (subsequent infusions): After the initial infusion rate, use either the 4-step process above or the following 5-step process: 3 mg/kg/hour, 6 mg/kg/hour, 8 mg/kg/hour, and then 10 mg/kg/hour; then, maintain the infusion rate at 10 mg/kg/hour until the infusion is complete. Approximate total 5-step infusion duration: 5 hours
• After the infusion is complete, flush the IV line with 5% Dextrose Injection.
• Discard any unused diluted product after 9 hours.
• Do not infuse this drug in the same IV line with other products.

• Lyophilized powder: Refrigerate vials at 2C to 8C (36F to 46F).
• Before reconstitution: Remove vials from the refrigerator and set aside for about 30 minutes to allow them to reach room temperature.
• Reconstituted solution: Should be diluted at once; if immediate use is not possible, can store up to 24 hours in a refrigerator (2C to 8C [36F to 46F]); do not freeze.
• Diluted solution: Should be administered at once; if immediate use is not possible, refrigerate at 2C to 8C (36F to 46F) for up to 24 hours; do not freeze.
• Completely infuse within 9 hours after removal from the refrigerator.
• If removed from the refrigerator, do not place back into the refrigerator.
• Discard if refrigerated more than 24 hours or if complete infusion is not possible within 9 hours after removal from the refrigerator.

• This drug must be reconstituted and diluted before use.
• The manufacturer product information should be consulted.

• Compatible: Sterile Water for Injection, USP; 5% Dextrose Injection

• General: Vital signs (more often during infusion in patients susceptible to fluid volume overload)

• Seek medical care if signs/symptoms of hypersensitivity reactions or IARs occur.
• Pregnant or lactating women exposed to this drug: You (or your health care provider) should report this exposure by calling 1-800-745-4447, extension 15500.
• The Pompe Registry has been established to better understand the variability and progression of Pompe disease, and to continue to monitor and evaluate long-term effects of this drug. Patients and their caregivers are encouraged to participate in the Pompe Registry; participation is voluntary and may involve long-term follow-up. For more information, visit www.registrynxt.com or call 1-800-745-4447, extension 15500.