Strong Ph III study data for BioMarin's PKU drug

The USA's BioMarin Pharmaceutical has reported positive final results from a Phase III trial of Kuvan (sapropterin). The data, which were published in the August 11 issue of The Lancet, suggest that treatment with Kuvan results in significant reductions in blood phenylalanine levels in some phenylketonuria patients. The investigational oral small-molecule drug for PKU, is being developed in partnership with German Swiss drugmaker Merck Serono.

Emil Kakkis, BioMarin's chief medical officer, said: "we recently received priority review from the [US Food and Drug Administration] and hope to receive FDA approval by late November. In the interim, we are pleased to be able to provide Kuvan to PKU patients prior to commercial availability through our expanded access program." The 89-patient, six-week study, found that the Kuvan cohort had a mean decrease in blood Phe levels of 236 uM/L (29%) versus an increase of 3 uM/L (3%) in the placebo group (p<0.0001). Prior to treatment, those in the Kuvan group and placebo arm had mean Phe levels of 843 uM/L and 888 uM/L, respectively, but after six weeks, 54% of treated patients and 23% of those on placebo had a concentration below the recommended 600 uM/L level (p=0.003).