Laronidase

Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis I (MPS I) is characterized by the deficiency of α-L-iduronidase, a lysosomal hydrolase which catalyzes the hydrolysis of terminal α-L-iduronic acid residues of dermatan sulfate and heparan sulfate.

• 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.

• None. None.contraindicated

For pretreatment recommendations, see Full Prescribing Information. ( 2.1 ) The recommended dosage is 0.58 mg/kg administered once weekly as an intravenous infusion. ( 2.2 ) For dosage and administration modifications due to hypersensitivity reactions or infusion-associated reactions (IARs), see Full Prescribing Information. ( 2.3 ) For instructions on preparation, storage, and administration, see Full Prescribing Information. ( 2.4 , 2.5 , 2.6 ) 2.1 Recommendations Prior to ALDURAZYME Treatment Premedication Prior to ALDURAZYME administration, consider premedicating with antihistamines, with or without antipyretics, 60 minutes before the start of infusion [see Warnings and Precautions (5.1 , 5.4) ]. Medical Support Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during ALDURAZYME administration. 2.2 Recommended Dosage and Administration The recommended dosage of ALDURAZYME is 0.58 mg/kg (actual body weight) administered once weekly as an intravenous infusion.

Risk of Acute Respiratory Complications: Patients with acute febrile or respiratory illness at the time of ALDURAZYME infusion may be at greater risk for infusion reactions. Consider delaying ALDURAZYME infusion. Sleep apnea is common in MPS I patients. Consider evaluating airway patency prior to initiation of treatment with ALDURAZYME. Appropriate respiratory support should be available during infusion. ( 5.2 ) Risk of Acute Cardiorespiratory Failure: Patients susceptible to fluid overload may be at increased risk for serious exacerbation of their cardiac or respiratory status during infusions. Consider a decreased total infusion volume and infusion rate when administering ALDURAZYME to these patients. Appropriate medical monitoring and support measures should be available during infusion. ( 2.2 , 5.3 ) Infusion Reactions: If severe IARs occur, discontinue ALDURAZYME and initiate appropriate medical treatment. ( 5.4 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Hypersensitivity reactions including anaphylaxis have been reported in patients during or up to 3 hours after ALDURAZYME infusions. Some of these reactions were life-threatening and included respiratory failure, respiratory distress, stridor, tachypnea, bronchospasm, obstructive airways disorder, hypoxia, hypotension, bradycardia, and urticaria. In clinical studies and postmarketing safety experience with ALDURAZYME, approximately 1% of patients experienced severe or serious hypersensitivity reactions.

Serious and or clinically significant adverse reactions described elsewhere in labeling include: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] Acute Respiratory Complications Associated with Administration [see Warnings and Precautions (5.2) ] Acute Cardiorespiratory Failure [see Warnings and Precautions (5.3) ] Infusion-Associated Reactions [see Warnings and Precautions (5.4) ] Most common adverse reactions (≥10%) in patients: 6 months of age and older are: infusion reactions (pyrexia, chills, blood pressure increased, tachycardia, and oxygen saturation decreased). ( 6.1 ) 6 years and older are: rash, upper respiratory tract infection, injection site reaction, hyperreflexia, paresthesia, flushing, and poor venous access. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genzyme at 1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice. Serious adverse reactions reported with ALDURAZYME treatment during clinical trials were anaphylactic and hypersensitivity reactions. The most common adverse reactions were infusion reactions. The frequency of infusion reactions decreased over time with continued use of ALDURAZYME, and the majority of reactions were classified as being mild to moderate in severity.

8.1 Pregnancy Pregnancy Exposure Registry An MPS I Registry has been established. Pregnant women with MPS I and healthcare providers are encouraged to contact the pregnancy sub-registry by visiting www.registrynxt.com or calling 1-800-745-4447 ext. 15500. Risk Summary Available data from the MPS I Registry pregnancy sub-registry, published case reports, and the global pharmacovigilance database with ALDURAZYME use in more than 30 pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. The continuation of treatment for MPS I during pregnancy should be individualized to the pregnant woman. Untreated MPS I may result in adverse pregnancy and infant outcomes ( see Clinical Considerations ). No evidence of fetal harm has been observed in rats when laronidase was administered during organogenesis at doses up to 6.2 times the recommended human dose (see Data ). The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

Metabolism

The pharmacokinetics of laronidase were evaluated in 6-year-old or older patients (N=10 to 12) with MPS I who received 0.58 mg/kg of body weight once weekly of ALDURAZYME as a 4-hour infusion in the placebo-controlled clinical study (Study 1). After the 1 st , 12 th , and 26 th weekly infusions, the mean maximum plasma concentrations (C max ) ranged from 1.2 to 1.7 mcg/mL for the 3 time points.

• Apr 30, 2003BLABLA125058Biomarin

1. 1Pyrexia50113%
2. 2Pneumonia2446.1%
3. 3Vomiting2025.1%
4. 4Dyspnoea1974.9%
5. 5Cough1834.6%
6. 6Malaise1834.6%
7. 7Fatigue1493.7%
8. 8Death1413.5%
9. 9Headache1313.3%
10. 10Influenza1283.2%
11. 11Condition Aggravated1243.1%
12. 12Pain1223.1%
13. 13Urticaria1183.0%
14. 14Inappropriate Schedule Of Product Administration1132.8%
15. 15Nasopharyngitis1042.6%

The 10 most recently updated of 21 ClinicalTrials.gov registrations naming Laronidase as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
  Recruiting · Phase 1 · Interventional · 10 enrolled · University of California, San Francisco
  NCT04532047updated 2026-03-17
• Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
  Active not recruiting · Observational · 13 enrolled · Masonic Cancer Center, University of Minnesota
  NCT05634512updated 2025-10-30
• China Post-marketing Surveillance (PMS) Study of Aldurazyme®
  Completed · Phase 4 · Interventional · 12 enrolled · Genzyme, a Sanofi Company
  NCT05134571updated 2025-09-17
• Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients
  Completed · Phase 3 · Interventional · 12 enrolled · Cinnagen
  NCT06406153updated 2024-08-06
• A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
  Terminated · Phase 4 · Interventional · 2 enrolled · Genzyme, a Sanofi Company
  NCT00418821updated 2024-02-20
• A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
  Completed · Observational · 57 enrolled · Sanofi
  NCT05073783updated 2024-02-14
• Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
  Terminated · Interventional · 6 enrolled · agnes chen
  NCT02232477updated 2023-10-31
• Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
  Completed · Phase 1 · Interventional · 11 enrolled · Masonic Cancer Center, University of Minnesota
  NCT01173016updated 2020-03-20
• Stem Cell Transplant w/Laronidase for Hurler
  Completed · Phase 2 · Interventional · 25 enrolled · Masonic Cancer Center, University of Minnesota
  NCT00176891updated 2019-07-11
• Intrathecal Enzyme Replacement for Hurler Syndrome
  Completed · Phase 1 · Interventional · 26 enrolled · Masonic Cancer Center, University of Minnesota
  NCT00638547updated 2018-12-03

Frequently asked questions

How does Laronidase work?

Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis I (MPS I) is characterized by the deficiency of α-L-iduronidase, a lysosomal hydrolase which catalyzes the hydrolysis of terminal α-L-iduronic acid residues of dermatan sulfate and heparan sulfate.

What is Laronidase used for?

According to FDA labeling, Laronidase carries indications including: 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.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Laronidase?

Laronidase is classified as Enzymes, Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme, Glycosaminoglycan Metabolism Increase, Increased Lysosomal Function.

What are the brand names for Laronidase?

Laronidase is marketed under brand names including Aldurazyme.

What are the contraindications for Laronidase?

Laronidase labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.