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🏅 FDA Orphan Designation

ALDURAZYME

LARONIDASE

Manufacturer: Genzyme Corporation

Indicated for:
Hurler syndromeOrphanHurler-Scheie syndromeOrphan
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Black Box Warning (Boxed Warning)

A Boxed Warning is the FDA's strongest safety warning — it indicates serious or life-threatening risks identified during clinical trials or post-market surveillance. It does not mean the medication is unsafe for all patients; your prescriber weighs these risks against the benefits for your specific situation. Source: FDA-approved prescribing information.

View full prescribing information on DailyMed (NLM) ↗

FDA-Approved Indications (2)

Hurler syndromeOrphan Designation

Patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and patients with the Scheie form who have moderate to severe symptoms. Reduces urinary glycosaminoglycans and improves

Hurler-Scheie syndromeOrphan Designation

Patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and patients with the Scheie form who have moderate to severe symptoms.

Indications & Usage

1 INDICATIONS AND USAGE ALDURAZYME ® is indicated for the treatment of: adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and patients with the Scheie form of MPS I who have moderate to severe symptoms. ALDURAZYME is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for the treatment of adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for the treatment of patients with the Scheie form of MPS I who have moderate to severe symptoms. ( 1 ) Limitations of Use: The risks and benefits of treating mildly affected patients with the Scheie form have not been established. ( 1 ) ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the disorder. ( 1 ) Limitations of Use The safety and effectiveness of treating mildly affected patients with the Scheie form have not been established. The effect of ALDURAZYME on central nervous system manifestations of the disorder has not been determined.

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ALDURAZYME
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Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.