Inborn errors of metabolism:
Indications for: JAVYGTOR
In conjunction with a phenylalanine (Phe)-restricted diet: to reduce blood Phe levels in hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU).
Adults and Children:
<1 month: not established. Take with food at same time each day. Tabs: swallow whole or may dissolve in 120–240mL water or apple juice (may crush tab) and drink within 15mins (tab may not completely dissolve). Powder: dissolve in 120–240mL water or apple juice (5mL if child weighs ≤10kg) and drink within 30mins. 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: initially 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 Phe levels after 1 week to assess effect, then periodically. Maintain Phe-restricted diet. Monitor for upper GI mucosal inflammation, hyperactivity. Discontinue if anaphylaxis occurs. Pregnancy. Nursing mothers.
Phenylalanine hydroxylase activator.
Monitor neurologic status with concomitant levodopa. Caution with drugs that can inhibit folate metabolism (eg, methotrexate, valproic acid, phenobarbital, trimethoprim). Concomitant drugs that affect nitric oxide-mediated vasorelaxation (eg, PDE-5 inhibitors); monitor BP.
Headache, rhinorrhea, pharyngolaryngeal pain, diarrhea, vomiting, cough, nasal congestion; hypersensitivity reactions, gastritis (monitor), hypophenylalaninemia.
Half-life: 6.7 hours (range: 3.9–17 hours).
Generic Drug Availability:
Tabs—30, 120, 500; Packets—1, 30