Version May 2024
Patients treated with cipaglucosidase alfa have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during cipaglucosidase alfa administration. If a severe hypersensitivity reaction (eg, anaphylaxis) occurs, cipaglucosidase alfa should be discontinued immediately, and appropriate medical treatment should be initiated. In patients with severe hypersensitivity reaction, desensitization measures to cipaglucosidase alfa may be considered.
Patients treated with cipaglucosidase alfa have experienced severe infusion-associated reactions (IARs). If severe IARs occur, immediately discontinue the cipaglucosidase alfa infusion, initiate appropriate medical treatment, and assess the benefits and risks of readministering cipaglucosidase alfa following severe IARs. Patients with an acute underlying illness at the time of cipaglucosidase alfa 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 a higher risk of severe complications from IARs.
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 cipaglucosidase alfa infusion. More frequent monitoring of vitals should be performed during cipaglucosidase alfa infusion in such patients.
Pompe disease (lysosomal acid alpha-glucosidase deficiency), late-onset:
Note: For use in combination with miglustat; initiate 2 weeks after last enzyme replacement therapy dose. Dose is based on actual body weight. Consider pretreatment with antihistamines, antipyretics, and/or corticosteroids; administer pretreatment in patients who received pretreatment with prior enzyme replacement therapy.
IV: 20 mg/kg infusion over ~4 hours every other week. Begin infusion ~1 hour after miglustat dose; infusion may be delayed for up to 3 hours after miglustat dose.
Missed dose:
If cipaglucosidase alfa infusion cannot be started within 3 hours of miglustat dose: Reschedule cipaglucosidase alfa and miglustat combination at least 24 hours after the last miglustat dose.
If both miglustat and cipaglucosidase alfa doses are missed: Restart treatment as soon as possible.
There are no dosage adjustments provided in the manufacturer's labeling.
There are no dosage adjustments provided in the manufacturer's labeling.
Hypersensitivity or infusion-associated reaction:
Mild to moderate hypersensitivity or moderate infusion-associated reaction: Temporarily hold or slow infusion rate.
If symptoms subside: Increase infusion rate to the rate at which the reaction occurred, then continue to increase the infusion rate every 30 minutes to the target infusion rate with close monitoring.
If symptoms persist: Stop infusion for 30 to 60 minutes; consider resuming infusion at a reduced rate if symptoms improve. If symptoms continue, discontinue infusion and reinitiate within 7 to 14 days with appropriate premedication.
Severe hypersensitivity or infusion-associated reaction: Discontinue infusion immediately and begin appropriate medical management. May consider rechallenge using slower infusion rates or desensitization measures. If tolerated, the infusion rate and dose may be increased to reach the usual recommended dose.
Refer to adult dosing.
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified. Reported adverse reactions are for combination therapy in adults.
>10%: Hypersensitivity: Hypersensitivity reaction (27%; anaphylaxis [3%], severe hypersensitivity reaction [3%]), infusion-related reaction (32%)
1% to 10%:
Cardiovascular: Flushing (2%), increased blood pressure (≥2%), tachycardia (2%; including sinus tachycardia)
Dermatologic: Skin rash (4%), urticaria (2%)
Gastrointestinal: Abdominal pain (≥5%), constipation (≥2%), diarrhea (6%), dysgeusia (2%), dyspepsia (≥2%), nausea (≥5%)
Hematologic & oncologic: Decreased platelet count (≥2%)
Local: Infusion-site reaction (swelling: ≥2%)
Nervous system: Asthenia (≥2%), chills (2%), fatigue (≥5%), headache (8%), malaise (≥2%), pain (≥2%), paresthesia (≥2%), tremor (≥2%)
Neuromuscular & skeletal: Arthralgia (≥2%), muscle spasm (2%), myalgia (≥2%)
Renal: Flank pain (≥2%)
Respiratory: Dyspnea (4%)
Miscellaneous: Fever (4%)
Frequency not defined: Immunologic: Antibody development (including neutralizing antibodies)
Pregnancy (when used in combination with miglustat).
Significant drug interactions exist, requiring dose/frequency adjustment or avoidance. Consult drug interactions database for more information.
Concerns related to adverse effects:
• Hypersensitivity: Cipaglucosidase alfa may cause severe hypersensitivity reactions, including severe infusion-associated reactions (eg, urticaria, pruritus, flushing) and anaphylaxis (some fatal). Ensure availability of appropriate medical support measures, including cardiopulmonary resuscitation equipment, during administration.
Disease-related concerns:
• Acute cardiorespiratory failure: 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 cipaglucosidase alfa infusion. Some patients may require prolonged observation times.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Solution Reconstituted, Intravenous:
Pombiliti: Cipaglucosidase alfa-atga 105 mg (1 ea) [contains polysorbate 80]
No
Solution (reconstituted) (Pombiliti Intravenous)
105 mg (per each): $2,142.00
Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.
IV: Inspect infusion bag for foaming prior to administration; if foaming is present, let foam dissipate before administering. If diluted solution has been refrigerated, allow to return to room temperature for 30 minutes prior to administration. Initiate cipaglucosidase alfa infusion ~1 hour after oral miglustat dose; if cipaglucosidase alfa infusion is delayed, starting infusion time should not exceed 3 hours after oral miglustat dose. Infuse through a separate line via an inline, low-protein-binding, 0.2-micron filter; change filter if the filter becomes blocked. Do not infuse in the same IV line as other products.
Initiate infusion at a rate of 1 mg/kg/hour. Gradually increase infusion rate by 2 mg/kg/hour every 30 minutes if no signs of hypersensitivity or infusion-associated reactions, up to a maximum rate of 7 mg/kg/hour. Then, maintain infusion rate at 7 mg/kg/hour until infusion is complete (total infusion duration: ~4 hours).
Pompe disease (lysosomal acid alpha-glucosidase deficiency), late-onset: Treatment of late-onset Pompe disease (lysosomal acid alpha-glucosidase deficiency), in combination with miglustat, in adults weighing ≥40 kg and who are not improving on enzyme replacement therapy.
None known.
There are no known significant interactions.
Evaluate pregnancy status prior to use; verify the patient is not pregnant prior to treatment initiation. Patients who may become pregnant should use effective contraception during therapy with cipaglucosidase alfa in combination with miglustat and for at least 60 days after the last dose.
Based on data from animal reproduction studies, in utero exposure to cipaglucosidase alfa in combination with miglustat may cause fetal harm. Use is contraindicated in patients who are pregnant.
It is not known if cipaglucosidase alfa is present in breast milk.
Due to the potential for serious adverse reactions in the breastfed infant, breastfeeding is not recommended by the manufacturer.
Hypersensitivity or infusion-associated reactions during infusion; frequent monitoring of vitals (during and immediately following infusion) in patients at risk for fluid volume overload (eg, underlying respiratory illness, compromised cardiac or respiratory function).
Pompe disease is caused by a deficiency of lysosomal acid alpha-glucosidase (GAA), which degrades glycogen in the lysosome. A deficiency of GAA results in accumulation of glycogen in various tissues. Cipaglucosidase alfa is an exogenous source of GAA, which results in degradation of intra-lysosomal glycogen.
Distribution: Vd: 2.0 to 4.7 L.
Metabolism: Expected to be metabolized into small peptides and amino acid via catabolic pathways.
Half-life elimination: 2.1 hours.
Excretion: Clearance: 0.8 L/hour.
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