Access program under way for new PKU drug

June 25, 2007

BioMarin Pharmaceutical and Merck Serono have announced the launch of their expanded-access program for sapropterin (Kuvan), an investigational agent for the treatment of phenylketonuria (PKU).

BioMarin Pharmaceutical and Merck Serono have announced the launch of their expanded-access program for sapropterin (Kuvan), an investigational agent for the treatment of phenylketonuria (PKU). The drug is the synthetic from of tetrahydrobiopterin, the naturally occurring enzyme cofactor that works with phenylalanine hydroxylase to metabolize phenylalanine. Under expanded access, BioMarin will provide the therapy at no cost to participating patients throughout the duration of the program. Patients must be over eight years old and have a confirmed diagnosis of PKU; at least 500 patients in the U.S. will be guaranteed enrollment in the program. According to the manufacturers, sapropterin has received orphan drug designation and, if granted priority review status, could receive FDA approval by the end of the year. For more information, call 1-(877) 811-7327.

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