North Bay pharmaceutical company BioMarin has reported that in a phase 3 PRISM-2 study of pegvaliase for phenylketonuria (PKU), preliminary data indicates that the drug met the primary goal of reducing blood phenylalanine in patients.
“We are committed to the global PKU community and to bringing them a medicine that has the potential to treat PKU adult patients,” said Hank Fuchs, M.D., Chief Medical Officer at BioMarin. “We look forward to sharing this data with the regulatory authorities in the US and Europe to continue the process of bringing an important therapy to patients.” BioMarin is a leader in developing and commercializing pharmaceuticals for rare genetic diseases. PKU is a disorder diagnosed in approximately 50,000 individuals worldwide.
To learn more, visit the BioMarin website.