Cipaglucosidase Alfa Dosage

Medically reviewed by Drugs.com. Last updated on Feb 8, 2024.

Usual Adult Dose for Pompe disease

At least 40 kg: 20 mg/kg IV every other week, infused over approximately 4 hours

• This drug must be given in combination with miglustat (Opfolda), an enzyme stabilizer.

Comments:

• Therapy with this drug may be started 2 weeks after the last dose of enzyme replacement therapy (ERT).
• Calculate the dose of this drug using the patient's actual body weight.
• Miglustat should be orally administered 1 hour before initiating infusion of this drug; refer to the manufacturer product information for miglustat for dosage and administration timeline instructions.

Use: In combination with miglustat, for the treatment of late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) in patients weighing 40 kg or greater and who are not improving on their current ERT.

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Dose Adjustments

MILD TO MODERATE HYPERSENSITIVITY REACTION OR MODERATE INFUSION-ASSOCIATED REACTION (IAR):

• Consider temporarily holding or slowing the infusion rate; initiate appropriate medical treatment.
• If symptoms subside after holding or slowing the infusion, increase the rate to the rate at which the reaction occurred and consider increasing the rate in a stepwise manner (every 30 minutes) to the target rate. Closely monitor the patient.
• If symptoms persist despite holding or slowing the infusion, stop the infusion for 30 to 60 minutes, monitor the patient, and consider resuming infusion at a reduced rate once the symptoms have improved.
• If symptoms continue to persist, then discontinue the infusion; consider reinitiating infusion within 7 to 14 days with appropriate premedication.

SEVERE HYPERSENSITIVITY REACTION (INCLUDING ANAPHYLAXIS) OR SEVERE IAR:

• Immediately discontinue infusion of this drug and institute appropriate medical treatment.
• Consider the risks and benefits of treatment re-administration after severe reactions.
• Patients may be rechallenged at a slower infusion rate; desensitization measures may be considered after severe hypersensitivity reaction.
• If this drug is readministered after a severe hypersensitivity reaction or IAR, ensure infusion tolerability before increasing the dosage (dose and/or rate) to reach the recommended dosage/infusion rate.

Precautions

US BOXED WARNINGS:

• HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Patients treated with this drug have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during administration of this drug. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, immediately discontinue this drug and initiate appropriate medical management. Desensitization measures may be considered in patients with severe hypersensitivity reactions.
• INFUSION ASSOCIATED REACTIONS (IARs): Patients treated with this drug have experienced severe IARs, and those with acute underlying illness at the time of infusion may be at greater risk. Patients with advanced Pompe disease may have compromised cardiac and respiratory function, predisposing these patients to a higher risk of severe complications from IARs. If severe IARs occur, immediately discontinue infusion of this drug, institute appropriate medical treatment, and assess the risk versus benefit of re-administration.
• RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients at risk of fluid volume overload or in those for whom fluid restriction is indicated due to acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of exacerbation of cardiac or respiratory status during infusion. Vitals should be monitored more frequently during infusion in these patients.

CONTRAINDICATIONS:

• Pregnancy

Safety and efficacy have not been established in patients younger than 18 years.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• Prior to starting treatment, verify the pregnancy status of females of childbearing potential.
• Before administration, consider pretreatment with antihistamines, antipyretics, and/or corticosteroids; in patients who utilized premedication with prior ERT regimens, pretreatment is recommended.
• Due to the potential for anaphylaxis, ensure appropriate medical support is readily available during administration.
• For IV use only; administer with inline low protein binding 0.2-micron filter; change filter if blockage occurs.
• Initiate IV infusion approximately 1 hour after oral administration of miglustat; do not administer this drug if the dose of miglustat was missed.
• If infusion of this drug cannot be started within 3 hours of oral administration of miglustat, reschedule administration of this drug (in combination with miglustat) to at least 24 hours after miglustat was last taken.
• If this drug in combination with miglustat is missed, restart treatment as soon as possible.

Infusion rate recommendations in adults weighing 40 kg or greater:

• Initial infusion rate: 1 mg/kg/hour
• Once tolerability is established, gradually increase rate by 2 mg/kg/hour every 30 minutes.
• Maximum infusion rate: 7 mg/kg/hour; maintain this rate until total infusion is completed (about 4 hours).
• See the manufacturer product information for weight-based infusion volumes and rates (mL/hour).

Storage requirements:

• Lyophilized powder: Store refrigerated at 2C to 8C (36F to 46F) in the original carton to protect from light.
• Reconstituted solution: If not used immediately, store refrigerated at 2C to 8C (36F to 46F) for up to 24 hours; do not freeze.
• Diluted solution:
• If not used immediately, store refrigerated at 2C to 8C (36F to 46F) for up to 16 hours; storage at room temperature is not recommended; do not freeze.
• Once removed from refrigeration, completely infuse within 6 hours; do not place back in refrigerator.
• Discard diluted solution if refrigerated longer than 16 hours or if unable to completely infuse within 6 hours after removal from refrigeration.

Reconstitution/preparation techniques:

• This drug must be reconstituted and diluted before use.
• Allow vial(s) to come to room temperature and reconstitute with Sterile Water for Injection; do not invert, shake, or swirl product.
• Further dilute reconstituted solution in 0.9% sodium chloride to a total infusion volume based on weight.
• The diluted infusion solution should come to room temperature (if previously refrigerated) before administration and be inspected for foaming; if foam is present, allow it to dissipate before infusing product.
• Consult the manufacturer product information for detailed instructions.

IV compatibility:

• Do not infuse with other products in the same IV line.

General:

• Do not use a pneumatic tube to transport the infusion bag containing the final product for administration.

Monitoring:

• Hypersensitivity: For signs/symptoms of hypersensitivity and infusion-associated reactions (during and after infusion)
• Metabolic: For fluid overload in patients at risk of exacerbating cardiac or respiratory status (during and after infusion)
• Respiratory: For signs/symptoms of acute underlying respiratory illness or compromised cardiac/respiratory function (prior to infusion)

Patient advice:

• Read the US FDA-approved patient labeling (if available).
• Follow the dosing timeline recommendations for administration of this drug (i.e., miglustat must be taken prior to infusion).
• Know the signs/symptoms of hypersensitivity reactions (including anaphylaxis) or infusion-associated, and seek immediate medical attention if reactions occur.
• Understand the increased risk of acute cardiorespiratory failure due to volume overload in patients with underlying respiratory illness or compromised cardiac or respiratory function.
• Lactating women: Breastfeeding is not recommended during use of this drug.
• Patients of childbearing potential:
• Notify health care provider of a known/suspected pregnancy.
• Use effective contraception during treatment and for at least 60 days after the last dose.
• This drug may impair male and female fertility.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

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