alpha-L-iduronidase

Orphan Drug Cold Chain RequiredFDA Approved, EMA Approved

Description

Aldurazyme is an enzyme replacement therapy for mucopolysaccharidosis I (MPS I). While primarily indicated for MPS I, it has been studied in related lysosomal storage disorders including fucosidosis due to similar pathophysiology. The recombinant enzyme helps break down glycosaminoglycans that accumulate in affected tissues.

Indications & Therapeutic Use

Mucopolysaccharidosis I, lysosomal storage disorders

Linked Diseases:

Global Availability (8 countries)

CountryAccess RouteRegulatory PathwayLead TimeStatus

Available Through

This drug is available for procurement through the following fulfilment partner.

Fulfilment Partner
alpha-L-iduronidase
Generic Namealpha-L-iduronidase
Brands1 brand available
Active Ingredientlaronidase
Drug ClassMucopolysaccharidosis I
ManufacturerBioMarin Pharmaceutical Inc.
Dosage FormsIV infusion, 0.58 mg/mL
Medical CodeA16AB05
Orphan StatusYes — Orphan Drug
Cold ChainRequired
Lead Time14 days
Reg. StatusFDA Approved, EMA Approved
Clinical TrialNCT00001203
Countries8 countries
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes