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Products

Aldurazyme® (laronidase) for MPS I

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. Aldurazyme is designed to address the underlying cause of the disease and provide the enzyme that people with MPS I are lacking.

Aldurazyme at a Glance

  • First specific approved therapy for the treatment of MPS I
  • Approved in multiple countries
  • Designated an orphan drug in the United States and internationally
  • Manufactured by BioMarin, commercialized by Genzyme Corporation

Regulatory Status

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 European Union, which grants the therapy market exclusivity for seven and 10 years, respectively. Aldurazyme has since been approved in numerous other countries around the world.

For additional information about Aldurazyme, please visit www.aldurazyme.com.

Commercialization Plan

Aldurazyme is manufactured by BioMarin and commercialized by Genzyme in the US, EU, and internationally.

Indication

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.


Important Safety Information

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.

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.
Patients with an acute illness at the time of ALDURAZYME infusion may be at greater risk for infusion-related reactions. Careful consideration should be given to the patient’s clinical status prior to administration of ALDURAZYME.


Patients should receive antipyretics and/or antihistamines prior to infusion. If an infusion reaction occurs, regardless of pretreatment, decreasing the infusion rate, temporarily stopping the infusion, and/or administration of additional antipyretics and/or antihistamines may ameliorate the symptoms.
The most common adverse reactions associated with ALDURAZYME treatment in the clinical studies were upper respiratory tract infection, rash, and injection site reaction The most common adverse reactions requiring intervention were infusion-related reactions involving flushing, fever, headache, and rash.


In postmarketing experience with ALDURAZYME, severe and serious infusion-related reactions have been reported, some of which were life-threatening. The most frequently reported adverse reactions included: chills, vomiting, nausea, arthralgia, diarrhea, tachycardia, abdominal pain, blood pressure increased, and oxygen saturation decreased.


Approximately 91% of patients treated with ALDURAZYME in clinical studies were positive for antibodies to laronidase. The clinical significance of antibodies to ALDURAZYME is not known, including the potential for product neutralization. Adverse events should be reported promptly to Genzyme Medical Information at 800-745-4447, option 2. ALDURAZYME is available by prescription only. To learn more, please see the full prescribing information including boxed warning, visit www.ALDURAZYME.com or contact Genzyme at 1-800-745-4447.