
ZURICH (Thomson Financial) - Biomarin Pharmaceutical and partner Merck Serono SA said phase III clinical tests show that their Kuvan drug reduced the level of phenylalanine (PHE) in the blood in some phenylketonuria (PKU) patients compared to a placebo.
Biomarin recently received priority review from the FDA for Kuvan, and hope to receive FDA approval by late November, said chief medical officer Emil Kakkis.
PKU is a genetic disorder caused by an enzyme deficiency which causes PHE to accumulate to abnormally high levels in the blood and becomes toxic to the brain.
It affects 50,000 diagnosed patients in the developed world.
The phase III test included 89 patients aged eight years and above in the US.
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