BioMarin Pharmaceutical Inc. announced that the US Food and Drug Administration (FDA) has accepted for filing and granted priority review status to the New Drug Application (NDA) for Kuvan (sapropterin dihydrochloride).
Under the Prescription Drug User Fee Act (PDUFA), the FDA is expected to take action on the application by November 25, 2007. Kuvan, an investigational oral small molecule for the treatment of phenylketonuria (PKU), a rare genetic metabolic disorder, is being developed in partnership with Merck Serono, a division of Merck KGaA, Darmstadt, Germany.
"We are pleased that Kuvan has been granted priority review status, a designation that emphasizes the significant need for an effective therapy to manage PKU. Over the next four months, we will work closely with the FDA to support their review of the NDA," said Jean-Jacques Bienaime, chief executive officer of BioMarin. "We are preparing to launch Kuvan in the US in December, if approved."
Priority review is typically granted to drugs that, if approved, will provide a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious or life-threatening disease. Kuvan has also been granted orphan drug designation, which is conferred upon investigational products for diseases that affect fewer than 200,000 patients in the United States. Products with orphan drug designation that are the first to be approved for a specific indication have seven years market exclusivity in the US.