Phenylketonuria
BioMarin announces positive results from phase III study of Phenoptin
Published in Drug Week, February 16th, 2007
Phenoptin is an investigational oral small molecule, being developed in partnership with Merck Serono, for the treatment of patients with phenylketonuria (PKU), who have elevated phenylalanine (Phe) levels. The results show that all pre-specified efficacy and safety endpoints of the double-blind, placebo-controlled study were met. Phenoptin treatment caused a significant increase in phenylalanine tolerance as well as a reduction in blood phenylalanine levels. In addition, the data...
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Source: Drug Week (2007-02-16)
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