CHMP recommends extending indication of Kuvan (sapropterin dihydrochloride) to children with phenylketonuria below 4 years of age-Merck Serono
Merck Serono has announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion on an update to the product information for its product Kuvan (sapropterin dihydrochloride), recommended that the Kuvan indication is extended to allow its use in children with phenylketonuria (PKU) below 4 years of age who have shown to be responsive to such treatment.
This is based on the SPARK Phase IIIb study which showed that the addition of Kuvan to a phenylalanine-restricted diet significantly increased phenylalanine tolerance by 30.5 mg/kg/day in children with PKU below 4 years of age and responsive to Kuvan, when compared with phenylalanine tolerance in children following a phenylalanine-restricted diet alone (p<0.001).
Comment: The original European marketing authorization for Kuvan was granted in 2008. In Europe, Kuvan is the only medication in combination with phenylalanine-restricted diet designed to reduce the concentration of phenylalanine in the blood and brain in those patients who are responsive to Kuvan to prevent the debilitating effects of PKU.
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