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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 |
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First specific approved therapy for the treatment of MPS I |
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Approved in multiple countries |
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Manufactured by BioMarin, commercialized by Genzyme Corporation (BioMarin/Genzyme LLC) |
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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.
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Important Safety Information |
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*Aldurazyme (laronidase)
is indicated for patients with Hurler and Hurler-Scheie
forms of 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 central nervous system manifestations
of the disorder.
The most common adverse reactions associated with Aldurazyme treatment in clinical studies were upper respiratory tract infection, rash, and injection site reaction. The most common adverse reactions requiring intervention were infusion-related reactions, including flushing, fever, headache, and rash. The most serious adverse reaction reported with Aldurazyme was an anaphylactic reaction, which occurred in one patient approximately three hours after the start of the infusion. The reaction consisted of urticaria and airway obstruction. Resuscitation required an emergency tracheostomy. This patient's pre-existing MPS I-related upper airway obstruction may have contributed to the severity of this reaction. Approximately 91 percent of patients treated with Aldurazyme were positive for antibodies to laronidase. The clinical significance of antibodies to Aldurazyme is not known. There are no known contraindications to the use of Aldurazyme. Aldurazyme is available by prescription only. For more information on Aldurazyme, please see full prescribing information at www.aldurazyme.com. |
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