laronidase

REMS

Mucopolysaccharidosis 1 (MPS 1; specifically Hurler and Hurler-Scheie form or Scheie form) with moderate to severe symptoms.

Contraind./Precautions ⬆ ⬇

Contraindicated in:

None.

Use Cautiously in:

OB: Safety not established in pregnancy;
Lactation: Safety not established in breastfeeding.

Adv. Reactions/Side Effects ⬆ ⬇

CV: edema

Derm: rash

Local: injection site reactions

Neuro: fatigue

Resp: respiratory tract infections

Misc: HYPERSENSITIVITY REACTIONS (INCLUDING ANAPHYLAXIS), infusion-related reactions

Interactions ⬆ ⬇

Drug-drug:

None reported.

Availability ⬆ ⬇

Solution for injection: 0.58 mg/mL

Route/Dosage ⬆ ⬇

IV (Adults and Children ): 0.58 mg/kg (actual body weight) once weekly.

US Brand Names ⬆ ⬇

Action ⬆ ⬇

Replaces the naturally occurring enzyme α-L-iduronidase, which is deficient in MPS 1. Without replacement, the glucosaminoglycans dermatan and heparan accumulate in tissues.

Therapeutic effects:

Decreased cellular, tissue, and organ damage due to mucopolysaccharide accumulation resulting in improved pulmonary function and walking capacity.

Classifications ⬆ ⬇

Therapeutic Classification: replacement enzyme

Pharmacologic Classification: enzymes

Pharmacokinetics ⬆ ⬇

Absorption: IV administration results in complete bioavailability.

Distribution: Unknown.

Metabolism/Excretion: Unknown.

Half-Life: 1.5–3.6 hr.

Time/Action Profile ⬆ ⬇

ROUTE	ONSET	PEAK	DURATION
IV	unknown	unknown	1 wk

Patient/Family Teaching ⬆ ⬇

Explain MPS 1 registry to patient and encourage participation. Purpose is to better understand the variability and progression of MPS 1 disease and to continue to monitor and evaluate treatments. Advise patients that participation may involve long-term follow-up. Information regarding the registry program can be found at https://www.mps1disease.com/patients/mpsi-registry.
Advise patient of risk of hypersensitivity reactions including anaphylaxis and infusion-related reactions. Caution patient to inform health care professional immediately if side effects occur.
Rep: Advise females of reproductive potential to notify health care professional if pregnancy is planned or suspected or if breastfeeding. Encourage pregnant patients to enroll in the Pregnancy Exposure Registry by visiting www.registrynxt.com or call 1-800-745-4447 ext. 15500.

Pronunciation ⬆ ⬇

Code ⬆

NDC Code

58468- Genzyme Corporation
- 58468-0070- ALDURAZYME
  - 58468-0070-1- 5 mL in 1 VIAL, GLASS (58468-0070-1)

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Indications ⬇