Aldurazyme® (laronidase) is an enzyme replacement therapy for the treatment of Mucopolysaccharidosis I (MPS I), an inherited, often life-threatening lysosomal disorder caused by a deficiency of the lysosomal enzyme, alpha-L-iduronidase. Lysosomes are part of the machinery for clearing waste and debris from the body’s cells. Aldurazyme is designed to address the underlying cause of the disease and provide the enzyme that people with MPS I are lacking.
In April 2003, Aldurazyme received marketing approval from the U.S. Food and Drug Administration making it the first specific therapy for the treatment of this progressive and debilitating disease. Shortly thereafter, in June 2003, the European Commission granted marketing authorization for Aldurazyme in the European Union. Aldurazyme has been designated orphan drug status in both the United States and the European Union. Aldurazyme has since been approved in numerous other countries around the world.
Aldurazyme is manufactured by BioMarin and commercialized by Genzyme in the US, EU, and internationally.
ALDURAZYME(laronidase) is indicated for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been established.
ALDURAZYME has been shown to improve pulmonary function and walking capacity. ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the disorder.
Life-threatening anaphylactic reactions have been observed in some patients during or up to 3 hours after ALDURAZYME infusions. Reactions have included: respiratory failure, respiratory distress, stridor, tachypnea, bronchospasm, airway obstruction, hypoxia, hypotension, bradycardia, and urticaria. Interventions have included: resuscitation, mechanical ventilatory support, emergency tracheotomy, hospitalization, and treatment with inhaled beta-adrenergic agonists, epinephrine, and intravenous corticosteroids.
In clinical trials and postmarketing safety experience with ALDURAZYME, approximately 1% of patients experienced severe or serious allergic reactions. In patients with MPS I, pre-existing upper airway obstruction may have contributed to the severity of some reactions. Due to the potential for severe allergic reactions, appropriate medical support should be readily available when ALDURAZYME is administered. Because of the potential for recurrent reactions, some patients who experience initial severe reactions may require prolonged observation. The risks and benefits of re-administering ALDURAZYME following an anaphylactic or severe allergic reaction should be considered.
WARNING
Risk of anaphylaxis. Life-threatening anaphylactic reactions have been observed in some patients during ALDURAZYME infusions. Therefore, appropriate medical support should be readily available when ALDURAZYME is administered. 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.
Generic Name - Laronidase solution for injection
2.9mg/ 5ml