The authorization follows the positive recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) in May this year, which was based on a review of data from a Phase IIIb SPARK trial.
The decision is applicable to all 28 member states in the European Union (EU) and the basis for corresponding decisions issued by Norway, Iceland and Liechtenstein.
Results from the 26-week SPARK trial showed that the addition of Kuvan to a phenylalanine-restricted diet significantly increased phenylalanine tolerance by 30.5mg/kg/day in children with PKU below four years of age and responsive to Kuvan, when compared with phenylalanine tolerance in children following a phenylalanine-restricted diet alone.
Merck Serono Global Research & Development head Luciano Rossetti said: "PKU is a rare disease with significant consequences – but if managed appropriately, it doesn’t have to impair child development or quality of life for children and adults.
"We are committed to helping patients with PKU, both at adult age and during childhood. The positive EC decision allows physicians to use Kuvan also in children right from diagnosis, who have shown to be responsive to the medication."
After securing EC approval, the Summary of Product Characteristics (SmPC) will be updated to include details about the use of Kuvan in this younger population.
Kuvan, which was jointly developed by BioMarin Pharmaceutical and Merck Serono, is the first oral therapy and approved in Europe to treat hyperphenylalaninemia (HPA).
Under the terms of the deal with BioMarin, Merck Serono has exclusive rights to market Kuvan in all territories outside the US, Canada and Japan.