Sapropterin for BH4 deficiency Initial and continuing PBS authority application 2025

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INDICATIONS AND USAGE Kuvan (sapropterin dihydrochloride) is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet.
Sapropterin is used to lower phenylalanine levels in the blood of patients with phenylketonuria (PKU). High levels of phenylalanine (an amino acid) in the blood can cause severe brain damage, including severe mental ation, seizures, tremors, or decreased learning ability.
Fluorouracil injection is indicated in the palliative management of some types of cancer, including colon, esophageal, gastric, rectum, , biliary tract, stomach, head and neck, cervical, pancreas, renal cell, and carcinoid. Fluorouracil is an antineoplastic anti-metabolite.
Esgic Capsules (butalbital, acetaminophen and caffeine capsules USP 50 mg/325 mg/40 mg) are indicated for the relief of the symptom complex of tension (or muscle contraction) headache.
INDICATIONS AND USAGE Natrecor (nesiritide) is indicated for the intravenous treatment of patients with acutely decompensated congestive heart failure who have dyspnea at rest or with minimal activity.

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BH4 deficiencies are more severe than PKU with regard to their response to therapy and treatment is substantially different. Low-Phe diet is not effective and early substitution with dopamine and serotonin precursors, as well as with the synthetic BH4 (sapropterin dihydrochloride) is crucial for a good outcome.
KUVAN (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution are prescription medicines used to lower blood Phe levels in adults and children over one month of age with a certain type of Phenylketonuria (PKU). KUVAN is to be used along with a Phe-restricted diet.

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