Generic Name and Formulations:
Sapropterin dihydrochloride 100mg; tabs.
In conjunction with a phenylalanine (Phe)-restricted diet: To reduce blood Phe levels in hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU).
<1 month: consult manufacturer. Take with food at same time each day. Dissolve tab in 4–8oz water or apple juice (may crush tab) and drink within 15 mins (tab may not completely dissolve). 1 month–6yrs: Initially 10mg/kg once daily; if inadequate response may increase to 20mg/kg once daily; if inadequate response after 1 month, discontinue. ≥7yrs: 10–20mg/kg once daily; if inadequate response after 1 month, discontinue. Usual range 5–20mg/kg per day; adjust based on blood Phe levels.
Monitor blood levels of Phe after 1 week to assess effect, then periodically. Discontinue if anaphylaxis occurs. Maintain Phe-restricted diet. Monitor BP and change in neurologic status. Hepatic or renal impairment; monitor. Pregnancy (Cat.C). Nursing mothers.
Caution with drugs that can inhibit folate metabolism (eg, methotrexate), PDE-5 inhibitors (eg, sildenafil, tadalafil, vardenafil), other nitric oxide donors, levodopa; monitor. May potentiate drugs that are P-gp or BCRP substrates.
Enzyme cofactor (BH4).
Headache, rhinorrhea, pharyngolaryngeal pain, diarrhea, vomiting, cough, nasal congestion, gastritis, hypophenylalaninemia, hyperactivity (monitor).
Tabs—120; Packet—1, 30
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