Extended US market exclusivity for BioMarin's rare disease drug Kuvan

US drugmaker BioMarin Pharmaceutical (Nasdaq: BMRN) says that the Food and Drug Administration has granted Kuvan (sapropterin dihydrochloride) powder for oral solution and tablets, used for the treatment of phenylketonuria (PKU), a six-month pediatric exclusivity extension.

The FDA action extends Kuvan’s market exclusivity to June 2015 and is based on studies submitted in response to a written request by the agency to investigate the use of the drug in pediatric patients from birth to age six. Kuvan is the first and only FDA-approved medication for PKU to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet.

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