FDA approval for Aldurazyme

BioMarin Pharmaceuticals from Novato, CA, and Genzyme General, Cambridge, MA, have announced that the FDA has granted marketing approval for Aldurazyme (laronidase), the first specific treatment approved for people with mucopolysaccharidosis I (MPS I).

Aldurazyme is indicated for patients with the Hurler and Hurler-Scheie forms of MPS I, and for Scheie patients with moderate to severe symptoms. 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.

Clinical trials have demonstrated that Aldurazyme can provide clinically important benefits for patients, including improved pulmonary function and walking ability. Aldurazyme has also been shown to be effective at reducing the excess carbohydrates that are stored in the organs of patients with MPS I, providing evidence that the enzyme is effective at a biochemical level.

As the first drug ever approved for MPS I, Aldurazyme has been granted orphan drug status in the United States, which confers seven years of market exclusivity. 'We have observed remarkable improvements in the lives of patients after treatment with Aldurazyme,' said Joseph Muenzer, MD, PhD, associate professor of Pediatrics, University of North Carolina, and a principal investigator for the Aldurazyme clinical trials. 'Prior to development of this enzyme replacement therapy, physicians were limited primarily to managing the wide array of symptoms in MPS I patients. Now, by addressing the underlying pathophysiology, we have the opportunity to change the course of this disease for the better.'