BioMarin Announces FDA Approval for Kuvan

First Specific Drug Therapy Approved for the Treatment of PKU

NOVATO, Calif., Dec. 13 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Kuvan(TM) (sapropterin dihydrochloride) Tablets, the first specific drug therapy approved for the treatment of phenylketonuria (PKU). Shipments to the distribution channel will commence tomorrow, and BioMarin will begin promotion of Kuvan immediately.

"The approval of Kuvan represents an important milestone for PKU patients and their families and also for BioMarin. We are extremely pleased to bring this promising treatment option to market in just a little over three years since the IND filing, and we are now ready for an immediate launch," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "We would like to thank all the patients, their families and physicians, our corporate partners, the FDA, and BioMarin employees for their hard work and dedication in making Kuvan a reality."

"In clinical trials, Kuvan has been shown to help control blood Phe levels in PKU patients, and I am thrilled that this new therapy is now commercially available to the PKU community," stated Dr. Barbara Burton, Professor of Pediatrics, Northwestern University Feinberg School of Medicine; Director, PKU Clinic at Children's Memorial Hospital; and Clinical Investigator in the Kuvan Phase 2 and Phase 3 trials. "With Kuvan now approved, physicians and patients have, for the first time, a drug therapy option to manage the disease."

Kuvan is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4) responsive PKU and is to be used in conjunction with a Phe-restricted diet. To determine if there is a response to Kuvan, the recommended starting dose of Kuvan is 10 mg/kg/day taken once daily for up to a month. If there is no response, the drug dose may be increased to 20 mg/kg/day for up to a month. The dose may be adjusted within a range of 5 to 20 mg/kg/day in patients who respond to Kuvan. Kuvan is developed in partnership with Merck Serono, a division of Merck KGaA, Darmstadt, Germany.