The Medical Letter on Drugs and Therapeutics
Sapropterin (Kuvan) for Phenylketonuria
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Sapropterin (Kuvan – BioMarin) has been approved by the FDA to reduce phenylalanine plasma concentrations in patients with phenylketonuria (PKU). PKU is a rare genetic disorder (it occurs in 1 in 8000 Caucasian and 1 in 50,000 African-American newborns) caused by a deficiency of phenylalanine hydroxylase (PAH); the resulting high levels of the amino acid in blood and urine lead to cognitive, behavioral and other neurological complications.1

MECHANISM OF ACTION — Sapropterin is a synthetic form of tetrahydrobiopterin (BH4), which is a cofactor in catabolism of phenylalanine. It acts by increasing PAH activity in patients who have residual enzyme function.

CLINICAL STUDIES — In an uncontrolled screening study, 489 patients with PKU who were 8-48 years old and not on a phenylalanine-restricted diet were treated with sapropterin 10 mg/kg/day for 8 days. On day 8, 96 ... more      

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Title: Sapropterin (Kuvan) for Phenylketonuria
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