Trade Names:Kuvan- Tablets 100 mg
Sapropterin is a synthetic form of tetrahydrobiopterin (BH4). Treatment with BH4 activates the residual phenylalanine hydroxylase enzyme, improves the normal oxidative metabolism of phenylalanine, and decreases phenylalanine levels in some patients.
The half-life in phenylketonuric (PKU) patients is approximately 6.7 h.
In PKU patients responsive to BH4, blood phenylalanine levels decrease within 24 h after a single dose; however, max effects on phenylalanine levels may take up to 1 mo.
Population pharmacokinetics did not differ in patients between 9 and 49 yr of age.
In conjunction with a phenylalanine-restricted diet, sapropterin reduces blood phenylalanine levels in patients with hyperphenylalaninemia due to BH4-responsive PKU.
PO Start with 10 mg/kg/day. If blood phenylalanine does not decrease from baseline, dose may be increased to 20 mg/kg/day after 1 month. Once responsiveness has been established, dose may be adjusted within range of 5 to 20 mg/kg/day. Discontinue treatment in patients whose blood phenylalanine does not decrease after 1 mo of treatment with 20 mg/kg/day. Doses above 20 mg/kg/day have not been evaluated.
Store at 59° to 86°F. Protect from moisture.
Additive effects may induce vasorelaxation, resulting in decreased BP.Drugs known to inhibit folate metabolism (eg, methotrexate)
Use with caution because the enzyme dihydropteridine reductase may be inhibited, resulting in decreased BH4 levels.Levodopa
Risk of adverse reactions, including convulsions, exacerbation of convulsions, irritability, or over-stimulation, may be increased.
None well documented.
Headache (15%); agitation, convulsions, dizziness, exacerbation of convulsions, irritability, overstimulation.
Rhinorrhea (11%); pharyngolaryngeal pain (10%); nasal congestion (4%).
Diarrhea, vomiting (8%); GI bleeding.
Increased gamma-glutamyltransferase (postmarketing).
Cough (7%); respiratory failure, upper respiratory tract infection.
Contusion (5%); peripheral edema, post-procedural bleeding.
Monitor blood phenylalanine levels to ensure adequate blood phenylalanine level control.
Category C .
Safety and efficacy not established in children younger than 4 yr of age.
Studies did not include patients older than 65 yr of age.
Carefully monitor use in patients with renal function impairment; use in these patients has not been evaluated.
Use with caution and close monitoring in patients with hepatic function impairment; treatment in these patients has not been evaluated.
Response to treatment can only be determined by a therapeutic trial.
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