laronidase
Orphan Drug Cold Chain RequiredFDA Approved, EMA Approved
Description
Laronidase is a recombinant form of human alpha-L-iduronidase used as enzyme replacement therapy. It is indicated for patients with Hurler and Hurler-Scheie forms of mucopolysaccharidosis I and for patients with Scheie form who have moderate to severe symptoms. While primarily used for MPS I, it has been studied in other lysosomal storage disorders with similar pathophysiology.
Indications & Therapeutic Use
Mucopolysaccharidosis I, lysosomal storage disorders
Global Availability (8 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
laronidase
| Generic Name | laronidase |
| Brands | 1 brand available |
| Active Ingredient | laronidase |
| Drug Class | Mucopolysaccharidosis I |
| Manufacturer | BioMarin Pharmaceutical Inc. |
| Dosage Forms | IV infusion, 0.58 mg/mL |
| Medical Code | A16AB05 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 14 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT00004454 |
| Countries | 8 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes