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sapropterin (Rx)Brand and Other Names:Kuvan

 
 
 

Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

tablet

  • 100mg

powder for oral solution

  • 100mg/packet
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Hyperphenylalaninemia

Indicated for hyperphenylalaninemia caused by tetrahydrobiopterin responsive phenylketonuria

10 mg/kg PO qDay initially with food; response to therapy is determined by change in blood Phe following treatment at 10 mg/kg/day for up to 1 month; blood Phe levels should be checked after 1 wk of therapy and periodically for up to a month; if blood Phe does not decrease from baseline at 10 mg/kg/day, the dose may be increased to 20 mg/kg/day; patients whose blood Phe does not decrease after 1 month of treatment at 20 mg/kg/day are non-responders and treatment should be discontinued 

Once responsiveness established, dosage may be adjusted within range of 5 to 20 mg/kg/day according to response to therapy; periodic blood Phe monitoring is recommended to assess blood Phe control

Dosing Considerations

Measure baseline blood Phe level before initiating therapy

If 20 mg/kg/day starting dose used, response to therapy is determined by change in blood Phe following therapy at 20 mg/kg/day for a period of 1 month; blood Phe levels should be checked after 1 wk of therapy and periodically during first month; treatment should be discontinued in patients who do not respond to therapy

Administration

Take with food

Tablet

  • Tablets may be swallowed either as whole tablets or dissolved
  • Dissolve in 4-8 oz (120-240 mL) water or apple juice and take within 15 minutes of dissolution
  • To make the tablets dissolve faster, stir or crush them
  • Tablets may not dissolve completely; small tablet pieces may float on top of the water or apple juice, this is normal and safe to swallow
  • If after drinking the medicine, add more water or apple juice to glass and drink to ensure the entire dose is taken

Powder for oral solution

  • Dissolved in 4-8 oz (120 to 240 mL) of water or apple juice and drink within 30 minutes of dissolution

Dosage Forms & Strengths

tablet

  • 100mg
more...

Hyperphenylalaninemia

Indicated for hyperphenylalaninemia caused by tetrahydrobiopterin responsive phenylketonuria

1 month to 6 years: Recommended starting dose is 10 mg/kg taken once daily

≥ 7 years: 10 mg/kg PO qDay initially with food; if 10 mg/kg/day starting dose used, response to therapy is determined by change in blood Phe following treatment at 10 mg/kg/day for up to 1 month; blood Phe levels should be checked after 1 wk of therapy and periodically for up to a month; if blood Phe does not decrease from baseline at 10 mg/kg/day, the dose may be increased to 20 mg/kg/day; patients whose blood Phe does not decrease after 1 month of treatment at 20 mg/kg/day are non-responders and treatment should be discontinued 

Once responsiveness established, dosage may be adjusted within range of 5-20 mg/kg/day according to response to therapy; periodic blood Phe monitoring is recommended to assess blood Phe control

Children aged <7 years treated with doses of 20 mg/kg/day are at increased risk for low levels of blood Phe

Dosing Considerations

Measure baseline blood Phe level before initiating therapy

Nonresponders: If 20 mg/kg/day starting dose used, response to therapy is determined by change in blood Phe following therapy at 20 mg/kg/day for a period of 1 month; blood Phe levels should be checked after 1 wk of therapy and periodically during first month; treatment should be discontinued in patients who do not respond to therapy

Administration

Take with food

Tablet

  • Tablets may be swallowed either as whole tablets or dissolved
  • Dissolve in 4-8 oz (120-240 mL) water or apple juice and take within 15 minutes of dissolution
  • To make the tablets dissolve faster, stir or crush them
  • Tablets may not dissolve completely; small tablet pieces may float on top of the water or apple juice, this is normal and safe to swallow
  • If after drinking the medicine, add more water or apple juice to glass and drink to ensure the entire dose is taken

Powder for oral solution

  • Dissolved in 4-8 oz (120 to 240 mL) of water or apple juice and drink within 30 minutes of dissolution
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Interactions

Interaction Checker

sapropterin and

No Results

     
     activity indicator 
    No Interactions Found
    Interactions Found

    Contraindicated

      Serious - Use Alternative

        Significant - Monitor Closely

          Minor

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            Adverse Effects

            >10%

            Rhinorrhea (11%)

            Headache (15%)

            1-10%

            Diarrhea (8%)

            Pharyngolaryngeal pain (10%)

            Cough (7%)

            Nasal congestion (4%)

            Vomiting (8%)

            Nausea (8%)

            <1%

            Gastritis

            Gastrointestinal bleeding

            Thrombocytopenia

            Spinal cord injury

            Abdominal pain

            Decreased appetite

            Respiratory tract infection

            Peripheral edema

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            Warnings

            Contraindications

            None listed in the manufacturer's label

            Cautions

            Do not eliminate ongoing Phe-restricted diet: treat all patients with a Phe-restricted diet

            Children < 7 years treated with 20 mg/kg/day are at increased risk for low levels of blood Phe compared with patients 7 years and older

            Treatment should be directed by physicians knowledgeable in management of PKU;. prolonged elevations in blood Phe levels in patients with PKU can result in severe neurologic damage, including severe mental retardation, microcephaly, delayed speech, seizures, and behavioral abnormalities; prolonged levels of blood Phe that are too low are associated with catabolism and protein breakdown; active management of dietary Phe intake while receiving therapy is required to ensure adequate Phe control and nutritional balance; monitor blood Phe levels during treatment to ensure adequate blood Phe level control; frequent blood monitoring is recommended in pediatric population

            Hypersensitivity reactions including anaphylaxis and rash have occurred

            Gastritis reported; monitor patients for signs and symptoms of gastritis

            Use caution in renal and hepatic impairment

            Patients that show no improvement after treating at 20 mg/kg/day for 1 month are considered nonresponders; response to treatment cannot be pre-determined by laboratory testing (e.g., molecular testing), and can only be determined by therapeutic trial

            Caution in coadministration of medications known to inhibit folate metabolism (eg, methotrexate), PDE-5 inhibitors, or levodopa

            Hyperactivity behavior reported (rare)

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            Pregnancy & Lactation

            Pregnancy Category: C

            Lactation: Excretion in breast milk unknown; not recommended

            Pregnancy Categories

            A:Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

            B:May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

            C:Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

            D:Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

            X:Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

            NA:Information not available.

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            Pharmacology

            Mechanism of Action

            Synthetic tetrahydrobiopterin (BH4), for phenylalanine hydroxylase (PAH); PAH hydroxylates Phe to form tyrosine; BH4 activates residual PAH enzyme, improving oxidative metabolism of Phe & thereby decreasing Phe blood levels

            Pharmacokinetics

            Half-Life: 7 hr; range: 3.9-17 hr

            Onset: 24 hr; up to 2 month (maximum effect)

            Peak Plasma Time: stable over 24-hr period

            Duration: 24 hr

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            Images

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            Formulary

            FormularyPatient Discounts

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            The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

            Tier Description
            1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
            2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
            3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
            4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
            5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
            6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
            NC NOT COVERED – Drugs that are not covered by the plan.
            Code Definition
            PA Prior Authorization
            Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
            QL Quantity Limits
            Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
            ST Step Therapy
            Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
            OR Other Restrictions
            Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
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