Biomarin develops drugs to treat rare genetic disorders. The company’s first marketed product is Aldurazyme, an enzyme replacement therapy for mucopolysaccharidosis I (MPS-I) that has an estimated prevalence of 3000 to 4000 patients worldwide. The second enzyme replacement product is Naglazyme for MPS-VI that BioMarin is selling in the US and Europe. The worldwide prevalence of MPS-VI is estimated at about 1000 patients.

Kuvan is an oral therapeutic on the market for phenylketonuria (PKU), an inherited metabolic disease with an estimated worldwide prevalence of 30 000 to 50 000. One of Biomarin’s lead product candidates in development is PEG-PAL, an enzyme substitution therapy for PKU with the potential to treat patients who do not respond to Kuvan. Initial data from a Phase II trial look promising and Phase III trials could begin at the end of 2011. The second key development candidate is GALNS (BMN-110) for Morquio A syndrome (MPS-IVA). Data from a Phase II trial were positive and we expect the start of a Phase III trial in the first quarter of 2011. To date, about 1000 patients with Morquio A syndrome have been identified and Biomarin estimates that there are at least 3000 patients worldwide, making GALNS Biomarin’s largest potential product to date.

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