Laronidase

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. Limitations of Use: The risks and benefits of treating mildly affected patients with the Scheie form have not been established. ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the disorder.

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. Reduced or absent α-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 I is to provide exogenous enzyme for uptake into lysosomes and increase the catabolism of GAG.

For pretreatment recommendations, see Full Prescribing Information. The recommended dosage is 0.58 mg/kg administered once weekly as an intravenous infusion. For dosage and administration modifications due to hypersensitivity reactions or infusion-associated reactions (IARs), see Full Prescribing Information. For instructions on preparation, storage, and administration, see Full Prescribing Information.

None. None.

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. 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. 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.

Serious and or clinically significant adverse reactions described elsewhere in labeling include: Hypersensitivity Reactions Including Anaphylaxis Acute Respiratory Complications Associated with Administration Acute Cardiorespiratory Failure Infusion-Associated Reactions 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 years and older are: rash, upper respiratory tract infection, injection site reaction, hyperreflexia, paresthesia, flushing, and poor venous access.

Refrigerate vials of ALDURAZYME at 2°C to 8°C (36°F to 46°F). Do not freeze or shake. Protect from light.