What is KUVAN®
KUVAN® (sapropterin dihydrochloride), in tablets or sachet for oral solution, is indicated for the treatment of hyperphenylalaninemia (HPA) in sapropterin-responsive adult and paediatric patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.
The rationale for KUVAN® administration in patients with BH4-responsive PKU is to enhance the activity of the defective phenylalanine hydroxylase (PAH), and thereby increase or restore the oxidative metabolism of Phe sufficient to reduce or maintain blood Phe levels, prevent or decrease further Phe accumulation, and increase tolerance to Phe intake in the diet.
The rationale for KUVAN® administration in patients with BH4 deficiency is to replace the deficient levels of BH4, thereby restoring the activity of PAH.
Mechanism of Action1
KUVAN® is a synthetic version of BH4, which is the naturally occurring cofactor essential for PAH enzyme function. In responsive patients, KUVAN® helps lower and maintain blood Phe levels, which may prevent neurotoxicity.