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news.BioMarin Pharmaceutical has released good results from a phase III extension study of Phenoptin, for the treatment of a genetic disorder called phenylketonuria.
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Phenoptin is for patients with phenylketonuria (PKU) who have elevated phenylalanine (Phe) levels. Preliminary data has suggested that Phenoptin has potential to produce significant reductions in blood Phe levels in the subset of patients who are BH4-responsive. BioMarin estimates that Phenoptin could be a potential treatment option for approximately 30% to 50% of individuals in the developed world who have PKU. Phenoptin has received orphan drug designation to treat PKU from both the FDA and European Medicines Agency.
Results confirm that all pre-specified safety and efficacy endpoints of the study were met. Phenoptin demonstrated an excellent safety and tolerability profile for all three doses, which was the primary endpoint of the extension study. A once daily dose regimen of Phenoptin was also sufficient to maintain the reduction of blood Phe levels throughout a 24 hour period.
An ongoing phase III diet study is designed to assess the increase in Phe tolerance levels in patients taking Phenoptin, and BioMarin said it remains on track to announce results in the first quarter of 2007.
In May 2005, BioMarin entered into a strategic partnership with Serono for the development and commercialization of Phenoptin. BioMarin and Serono expect to file for approval in the second and third quarters of 2007.