Phenylketonuria (PKU) is a genetic disorder that is characterized by an inability of the body to utilize the essential amino acid, phenylalanine. Phenoptin™ (sapropterin dihydrochloride) is an investigational oral small molecule for the treatment of phenylketonuria (PKU). Today, BioMarin Pharmaceutical Inc. and Serono announced the positive results of Phase 3, double-blind, placebo-controlled clinical study of Phenoptin™.
Results confirm that all pre-specified primary and secondary endpoints were met and data from the Phase 3 study demonstrate a statistically significant reduction at six weeks in blood phenylalanine (Phe) levels (p<0.0001) in patients receiving Phenoptin, compared with those receiving placebo.
Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin stated, “This is an exciting day for PKU patients worldwide as a simple oral treatment that could potentially help them reach their treatment goals is now one step closer to becoming a reality. For many patients with genetic diseases, not enough has been done to push forward important new treatments, and we are particularly happy to be able to collaborate with the numerous clinicians and investigators worldwide who have worked on tetrahydrobiopterin and transform their hard work into what could become the first approved drug therapy for this disease.”
Read Serono’s full press release.
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