Usual Adult Dose for Pompe disease

20 mg/kg via IV infusion every 2 weeks

Comments:

• Total volume of infusion is determined by patient's body weight and should be infused over about 4 hours.

Usual Pediatric Dose for Pompe disease

20 mg/kg via IV infusion every 2 weeks

Comments:

• Total volume of infusion is determined by body weight and should be infused over about 4 hours.

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Precautions

US BOXED WARNINGS:
RISK OF ANAPHYLAXIS, HYPERSENSITIVITY, AND IMMUNE-MEDIATED REACTIONS:

• Life-threatening anaphylactic reactions and severe hypersensitivity reactions presenting as respiratory distress, hypoxia, apnea, dyspnea, bradycardia, tachycardia, bronchospasm, throat tightness, hypotension, angioedema (including tongue or lip swelling, periorbital edema, and face edema), and urticaria observed in some patients during and after drug infusion.
• Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions reported in some patients after treatment.
• Patients should be observed closely during and after drug infusion and healthcare providers should be prepared to manage anaphylaxis and hypersensitivity reactions; appropriate medical support should be readily available when this drug is used.
• Patients should be informed of the signs/symptoms of anaphylaxis, hypersensitivity reactions, and immune-mediated reactions and should be instructed to seek immediate medical care if signs/symptoms occur.

• Infantile-onset Pompe disease patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbations of their cardiac or respiratory compromise due to fluid overload or infusion reactions; additional monitoring is recommended.

Dialysis

Data not available

Other Comments

Administration advice:

• Administer infusions in a stepwise manner using an infusion pump and an in-line low protein-binding 0.2 micrometer filter.
• Initial infusion rate should not exceed 1 mg/kg/hr; may increase in increments of 2 mg/kg/hr every 30 minutes, after tolerance to infusion rate established, up to maximum rate of 7 mg/kg/hr; obtain vital signs at the end of each step. If patient is stable, administer at maximum rate of 7 mg/kg/hr until infusion is completed.
• Slow or temporarily stop infusion if mild/moderate hypersensitivity reaction or infusion reaction; if anaphylaxis or severe hypersensitivity reaction, immediately discontinue drug and start appropriate medical treatment.
• Consult the manufacturer product information for recommended infusion volumes and rates.

• Store vials of lyophilized cake/powder in refrigerator at 2C to 8C (36F to 46F).
• Reconstituted and diluted solution: Stable for up to 24 hours at 2C to 8C (36F to 46F) if immediate use not possible; protect from light. Reconstituted solution should not be stored at room temperature.
• Do not freeze or shake.

• The manufacturer product information should be consulted.
• Final concentration: 0.5 to 4 mg/mL

• Compatible with Sterile Water for Injection, USP and 0.9% Sodium Chloride for Injection, USP
• Should not be infused in the same IV line with other products

• This drug should be reconstituted, diluted, and administered by a healthcare professional.

• General: Vital signs (at the end of each step during administration); for infusion reactions (during and up to 2 hours after infusion)
• Genitourinary: Urinalysis (periodically)
• Hypersensitivity: For signs/symptoms of systemic immune-mediated reactions involving skin and other organs (during therapy)
• Immunologic: For IgG antibody formation (every 3 months for 2 years and annually thereafter); for IgG titers if hypersensitivity reactions, other immune-mediated reactions; for IgG titers and inhibitory antibody activity in patients with reduced clinical response; for IgE antibodies to alglucosidase alfa or other mediators of anaphylaxis if anaphylactic or hypersensitivity reactions

• If signs/symptoms of anaphylaxis, hypersensitivity reactions, or immune-mediated reactions occur, seek medical care.
• A voluntary Pompe Registry has been established. For additional information and enrollment: pomperegistry.com