alglucosidase

REMS

MyozymeReplacement enzyme in infantile-onset Pompe disease (alpha glucosidase [GAA] deficiency).
LumizymeReplacement enzyme in late-onset and infantile onset Pompe disease.

Contraind./Precautions ⬆ ⬇

Contraindicated in:

None known.

Use Cautiously in:

Acute underlying illness (↑ risk of infusion reactions);
OB: Use during pregnancy only if potential maternal benefit justifies potential fetal risk;
Lactation: Use while breastfeeding only if potential maternal benefit justifies potential risk to infant;
Pedi: Safety not established in children <1 mo or >3.5 yr (Myozyme).

Adv. Reactions/Side Effects ⬆ ⬇

CV: bradycardia, tachycardia, edema

Derm: dermatitis, flushing, rash, urticaria, NECROTIZING SKIN LESIONS

EENT: blurred vision, vertigo

GI: constipation, diarrhea, reflux, vomiting

GU: nephrotic syndrome, proteinuria

Hemat: anemia, lymphadenopathy

Resp: ↓oxygen saturation, cough, respiratory failure, tachypnea

Misc: fever, HYPERSENSITIVITY REACTIONS (INCLUDING ANAPHYLAXIS), INFUSION REACTIONS

Interactions ⬆ ⬇

Drug-drug:

None reported.

Availability ⬆ ⬇

Lyophilized powder for injection: 50 mg/vial

Route/Dosage ⬆ ⬇

IV (Children 1 mo–3.5 yr): Myozyme: 20 mg/kg every 2 wk.
IV (Adults and Children ): Lumizyme: 20 mg/kg every 2 wk.

US Brand Names ⬆ ⬇

Action ⬆ ⬇

Replaces alpha-glucosidase. 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 survival with delayed need for ventilatory support.
Improved lung function and exercise capacity.

Classifications ⬆ ⬇

Therapeutic Classification: replacement enzyme

Pharmacokinetics ⬆ ⬇

Absorption: IV administration results in complete bioavailability.

Distribution: Unknown.

Metabolism/Excretion: Unknown.

Half-Life: 2.3–2.5 hr.

Time/Action Profile ⬆ ⬇

ROUTE	ONSET	PEAK	DURATION
IV	unknown	end of infusion	2 wk

Patient/Family Teaching ⬆ ⬇

Explain purpose of alglucosidase to patient and caregiver.
Advise patient to notify health care professional immediately if signs and symptoms of hypersensitivity reaction or cardiorespiratory failure occur.
Rep: Advise females of reproductive potential to notify health care professional if pregnancy is planned or suspected and to avoid breastfeeding during and for 24 hr after last dose. Encourage females of reproductive potential to enroll in registry for patients with Pompe disease to monitor the effect of alglucosidase on pregnant women and their offspring. For information, visit www.registrynxt.com or call 1-800-745-4447 ext. 15500.

section name header

Indications ⬇