sapropterin

Contraindicated in:

Hypersensitivity;
Lactation: Lactation.

Use Cautiously in:

Hepatic or renal impairment;
Concurrent use of levodopa;
OB: Use during pregnancy only if potential maternal benefit justifies potential fetal risk;
Pedi: Children <4 yr (safety and effectiveness not established).

Adv. Reactions/Side Effects ⬆ ⬇

Derm: rash

EENT: pharyngolaryngeal pain, rhinitis

GI: abdominal pain, diarrhea, esophagitis, gastritis, nausea, vomiting

Hemat: neutropenia

Neuro: headache, hyperactivity

Misc: HYPERSENSITIVITY REACTIONS (INCLUDING ANAPHYLAXIS)

Interactions ⬆ ⬇

Drug-drug:

Concurrent use of medications known to inhibit folate metabolism including methotrexate can ↓ BH4 levels; use cautiously.
Concurrent use of medications known to affect nitric oxide-mediated vasorelaxation including avanafil, sildenafil, vardenafil , or tadalafil could ↑ risk of hypotension.
Concurrent use of levodopa may ↑ risk of seizures, over-stimulation and irritability; use cautiously.

Availability ⬆ ⬇

(Generic available)

Powder for oral solution: 100 mg/pkt; 500 mg/pkt
Tablets: 100 mg

Route/Dosage ⬆ ⬇

PO (Adults and Children ≥7 yr): 10–20 mg/kg once daily, titrated on the basis of Phe levels (range 5–20 mg/kg/day).
PO (Children 1 mo-6 yr): 10 mg/kg once daily, titrated on the basis of Phe levels (range 5–20 mg/kg/day).

US Brand Names ⬆ ⬇

Action ⬆ ⬇

Acts as a synthetic form of the cofactor (BH4) for the enzyme phenylalanine hydoxylase (PAH). PAH converts phenylalanine to tyrosine. In PKU patients, activity of PAH is deficient. BH4 helps to activate PAH and thus reduce Phe levels.

Therapeutic effects:

Preservation of brain function by lowering Phe levels.

Classifications ⬆ ⬇

Therapeutic Classification: antihyperphenylalaninemics

Pharmacologic Classification: synthetic BH4

Pharmacokinetics ⬆ ⬇

Absorption: Well absorbed following oral administration; food ↑ absorption.

Distribution: Unknown.

Metabolism/Excretion: Unknown.

Half-Life: 6.7 hr.

Time/Action Profile ⬆ ⬇

(effect on Phe levels)

ROUTE	ONSET	PEAK	DURATION
PO	within 24 hr	up to 1 mo	unknown

Patient/Family Teaching ⬆ ⬇

Instruct patient to take sapropterin as directed at the same time each day. Take missed doses as soon as remembered that day; do not take 2 doses in the same day, omit dose if remembered next day. Instruct patient to read the Patient Information guide prior to taking sapropterin and with each Rx refill, in case of new information.
Advise patient to avoid making changes to dietary PhE without consulting health care professional; any dietary changes may affect Phe level.
Advise patient to notify health care professional if signs and symptoms of gastritis (severe upper stomach-area (abdominal) discomfort or pain, nausea, vomiting, blood in vomit or stool, black, tarry stools) or hyperactivity (fidgeting, moving around too much, talking too much) occur.
Instruct patient to notify health care professional if fever or illness occurs; dose may need to be adjusted.
Instruct patient to notify health care professional of all Rx or OTC medications, vitamins, or herbal products being taken and to consult with health care professional before taking other medications.
Rep: Advise females of reproductive potential to notify health care professional if pregnancy is planned or suspected or if breastfeeding. To reduce risk of hyperphenylalaninemia-induced fetal adverse effects, maintain blood phenylalanine concentrations between 120 and 360 micromol/L during pregnancy and during the 3 mo before conception. Advise women who become pregnant while taking sapropterin to enroll in Pregnancy Registry to monitor pregnancy outcomes. For more information about the registry program call 1-800-983-4587.

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Indications ⬇

Contraind./Precautions ⬆ ⬇