RxTrials Institute Drug Pipeline Alert

After receiving promising findings from two Phase III trials, a committee of the European Medicines Agency issued a positive opinion for Merck Serono’s Kuvan as an oral treatment for hyperphenylalaninemia in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.

PKU and BH4 deficiency are rare diseases caused by genetic defects in the metabolism of the amino acid phenylalanine, resulting in hyperphenylalaninemia, or abnormally high levels of phenylalanine in the blood.

Data from two international, double-blind, randomized, placebo-controlled trials in patients with hyperphenylalaninemia due to PKU show that treatment with the drug reduced blood phenylalanine levels and increased dietary phenylalanine tolerance, Merck said.