cipaglucosidase alfa

Replaces lysosomal acid alpha-glucosidase, which is deficient in Pompe disease. Without this enzyme, glycogen accumulates in tissues, including cardiac and skeletal muscles and hepatic tissues, leading to the development of cardiomyopathy, progressive muscle weakness, and impairment of respiratory function.

Therapeutic effects:

Improved lung function and exercise capacity.

Pharmacokinetics ⬆ ⬇

Absorption: IV administration results in complete bioavailability.

Distribution: Minimally distributed to tissues.

Metabolism/Excretion: Metabolized into small peptides and amino acids via catabolic pathways. Excretion pathway unknown.

Half-Life: 2.1 hr.

Time/Action Profile ⬆ ⬇

(plasma concentrations)

ROUTE	ONSET	PEAK	DURATION
IV	rapid	end of infusion	unknown

Contraind./Precautions ⬆ ⬇

Contraindicated in:

OB: Pregnancy;
Lactation: Lactation.

Use Cautiously in:

Acute underlying illness (↑ risk of infusion-associated reactions);
Susceptible to volume overload, or with acute underlying respiratory illness or compromised cardiac or respiratory function when fluid restriction is indicated (↑ risk of worsening cardiac or respiratory function);
Rep: Women of reproductive potential;
Pedi: Safety and effectiveness not established in children.

Adv. Reactions/Side Effects ⬆ ⬇

CV: hypertension, hypotension, tachycardia

Derm: flushing, pruritus, rash, urticaria

GI: abdominal distention, abdominal pain, constipation, diarrhea, dyspepsia, nausea

GU: ↓fertility

Hemat: thrombocytopenia

Local: infusion site swelling

MS: arthralgia, muscle spasms, myalgia

Neuro: fatigue, dizziness, dysgeusia, headache, paresthesia, sedation, tremor

Resp: dyspnea

Misc: hypersensitivity reactions (including anaphylaxis), infusion-associated reactions, chills, fever

Interactions ⬆ ⬇

Drug-drug:

None reported.

Route/Dosage ⬆ ⬇

IV (Adults ): 20 mg/kg (actual body weight) every other wk. Initiate infusion 1 hr after administration of miglustat.

Availability ⬆ ⬇

Lyophilized powder for injection: 105 mg/vial

Assessment ⬆ ⬇

Assess vitals signs and for underlying respiratory illness, difficulty breathing, compromised cardiac status (fluid volume overload) during and after infusions.
Assess for hypersensitivity reactions (hives, itching, swelling, flushing, hypotension, tachycardia, dizziness). If a severe hypersensitivity reaction (anaphylaxis) occurs, discontinue immediately, and initiate appropriate medical treatment (epinephrine).
Assess for infusion-associated reactions (urticaria, pruritus, flushing). If mild to moderate hypersensitivity or moderate infusion-associated reaction reaction occurs, stop the infusion for 30–60 min. Monitor the patient, and resume the infusion at a ↓ rate if symptoms have improved. If symptoms persist, discontinue the infusion, and consider reinitiating the infusion within 7–14 days with appropriate premedication. If symptoms subside following holding or slowing the infusion,↑ infusion rate to the rate at which the reaction occurred and consider continuing to ↑ the rate (every 30 min) in a stepwise manner up to the target infusion rate. Closely monitor the patient.

Lab Test Considerations:

Verify negative pregnancy test before starting therapy.

Implementation ⬆ ⬇

Administer in a health care setting with appropriate medical monitoring and supportive measures (cardiopulmonary resuscitation equipment) available during administration.
Administer antihistamines, antipyretics, and/or corticosteroids before infusion, depending on individual status.
Initiate the cipaglucosidase infusion approximately 1 hr after oral administration of miglustat. If the infusion cannot be started within 3 hr of oral miglustat, reschedule ≥24 hr after miglustat was last taken. If cipaglucosidase in combination with miglustat are both missed, restart treatment as soon as possible.

IV Administration:

Y-Site Incompatibility: Do not administer other drugs through same IV line.

Patient/Family Teaching ⬆ ⬇

Explain purpose and side effects of cipaglucosidase to patient. Advise patient to read Patient Information before starting therapy. Advise the patient to follow the time line recommendations for taking miglustat before the IV infusion.
Advise patient to notify health care professional of all Rx or OTC medications, vitamins, or herbal products being taken and to consult health care professional before taking other medications.
Teach patient the signs and symptoms of an hypersensitivity reaction; if any occur, seek immediate medical care.
Instruct patient to notify health care professional of any signs and symptoms of an infusion-associated reaction.
Advise patient to notify a health care professional if they are experiencing respiratory illness or compromised cardiac symptoms (volume overload) during therapy.
Rep: May cause fetal harm. Advise females of reproductive potential to use effective contraception during treatment and for ≥60 days after the last dose. Advise females not to breastfeed. Advise males or females of reproductive potential that treatment may impair fertility.

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Pronunciation ⬇

Classifications ⬆ ⬇

Indications ⬆ ⬇

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