Proper name: Laronidase
Manufacturer: Biomarin Pharmaceutical Inc, Novato, CA, License #1649
Indication for Use: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. Laronidase has been shown to improve pulmonary function and walking capacity. Laronidase has not been evaluated for effects on the central nervous system manifestations of the disorder.
Approval Date: 4/30/2003
Type of submission: Biologics license application