Kuvan®
Kuvan® (sapropterin dihydrochloride) is an oral drug therapy indicated for the treatment of hyperphenylalaninemia (HPA) in adults and children of 4 years of age and over with PKU or BH4 deficiency who have been shown to be responsive to such treatment. HPA can cause serious brain damage in infants and children, and transient to lasting neurocognitive impairment in adult patients.
Clinical data suggests that Kuvan® produces significant reductions in blood phenylalanine (Phe) levels among patients who are BH4 responsive. Kuvan allows individuals with HPA to optimize control of Phe levels, helping them to achieve treatment targets.
Kuvan® is the first and only approved pharmaceutical formulation of BH4 in Europe. BH4 is the cofactor required for the metabolism of Phe by the enzyme phenylalanine hydroxylase (PAH). Kuvan effectively treats the underlying pathophysiology of PKU by stimulating the activity of the residual PAH enzyme in patients who are responsive to BH4 treatment.
A synthetic form of BH4 that can be taken orally,Kuvan®is provided as soluble tablets, administered once daily.
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