Last reviewed · How we verify
Aldurazyme (LARONIDASE)
Aldurazyme breaks down abnormal sugar molecules in the body to treat Mucopolysaccharidosis I.
Aldurazyme (Laronidase) is a hydrolytic lysosomal glycosaminoglycan-specific enzyme developed by Biogen (not Biomarin, though it was acquired by them) and approved by the FDA in 2003. It is a small molecule modality used to treat Mucopolysaccharidosis I (MPS-I), a rare genetic disorder. The commercial status of Aldurazyme is patented, and it is currently owned by Biogen. Key safety considerations include potential infusion reactions and the risk of anaphylaxis. Aldurazyme works by breaking down abnormal sugar molecules in the body.
At a glance
| Generic name | LARONIDASE |
|---|---|
| Sponsor | BioMarin |
| Drug class | Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme [EPC] |
| Modality | Enzyme |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 2003 |
| Annual revenue | 300 |
Mechanism of action
Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis (MPS I) is characterized by the deficiency of -L-iduronidase, lysosomal hydrolase which catalyzes the hydrolysis of terminal -L-iduronic acid residues of dermatan sulfate and heparan sulfate. Reduced or absent -L-iduronidase activity results in the accumulation of the GAG substrates, dermatan sulfate and heparan sulfate, throughout the body and leads to widespread cellular, tissue, and organ dysfunction.The rationale of ALDURAZYME therapy in MPS is to provide exogenous enzyme for uptake into lysosomes and increase the catabolism of GAG. ALDURAZYME uptake by cells into lysosomes is most likely mediated by the mannose-6-phosphate-terminated oligosaccharide chains of laronidase binding to specific mannose-6-phosphate receptors. Because many proteins in the blood are restricted from entry int
Approved indications
- Mucopolysaccharidosis, MPS-I
Boxed warnings
- WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, and ACUTE RESPIRATORY COMPLICATIONS ASSOCIATED WITH ADMINISTRATION WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, and ACUTE RESPIRATORY COMPLICATIONS ASSOCIATED WITH ADMINISTRATION See full prescribing information for complete boxed warning . Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, discontinue ALDURAZYME immediately and initiate appropriate medical treatment. ( 5.1 ) Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions and require additional monitoring. Appropriate respiratory support should be available during infusion. ( 5.2 ) Hypersensitivity Reactions Including Anaphylaxis Patients treated with ALDURAZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during ALDURAZYME administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ALDURAZYME immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reactions, a desensitization procedure to ALDURAZYME may be considered [see Warnings and Precautions (5.1) ] . Acute Respiratory Complications Associated with Administration Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions and require additional monitoring [see Warnings and Precautions (5.2) ] .
Common side effects
- Infusion reactions
- Rash
- Upper respiratory tract infection
- Pyrexia
- Chills
- Oxygen saturation decreased
- Tachycardia
- Blood pressure increased
- Hyperreflexia
- Paresthesia
- Poor venous access
- Injection site reaction
Key clinical trials
- PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) (PHASE1)
- Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
- China Post-marketing Surveillance (PMS) Study of Aldurazyme® (PHASE4)
- Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients (PHASE3)
- A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants (PHASE4)
- 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
- Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I (NA)
- Administration of IV Laronidase Post Bone Marrow Transplant in Hurler (PHASE1)
Patents
| Patent | Expiry | Type |
|---|---|---|
| Biologic Exclusivity |
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Aldurazyme CI brief — competitive landscape report
- Aldurazyme updates RSS · CI watch RSS
- BioMarin portfolio CI