What is Sapropterin used for?
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 retardation, seizures, tremors, or decreased learning ability.
What is Kuvan used for?
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.
What is KUVAN made of?
BH4 is a natural substance found in the body that helps the PAH enzyme reduce Phe to safe levels in the blood. The active ingredient in KUVAN® (sapropterin dihydrochloride) Tablets or Powder for Oral Solution is a pharmaceutical version of BH4. It works in the same way as your body’s BH4.
How does KUVAN help PKU?
How does KUVAN work? In many people with PKU, KUVAN increases the activity of the PAH enzyme that isn’t working properly. In other words, the PAH enzyme that isn’t working “wakes up” and starts to process the Phe. This helps to lower the amount of Phe in the blood.
Is Hyperphenylalaninemia the same as PKU?
Signs. Benign hyperphenylalaninemia (H-PHE) is a form of phenylketonuria (PKU). Different forms of PKU have varying severity of signs. Because H-PHE is a less severe type of PKU, babies with H-PHE typically do not show any signs.
What is Dihydropteridine reductase?
Dihydropteridine reductase deficiency (DHPR) is a severe form of hyperphenylalaninemia (high levels of the amino acid phenylalanine in the blood) due to impaired renewal of a substance known as tetrahydrobiopterin (BH4).
How is tyrosine made?
Tyrosine is a nonessential amino acid and can be formed by the hydroxylation of phenylalanine in the liver when the intake of tyrosine in the diet is low.
Who can take KUVAN?
KUVAN: Proven and Effective
- In addition to adults, children and teens with PKU, aged 1 month to 16 years, have been treated with KUVAN in clinical studies.
- Children younger than 7 years old taking KUVAN are at a higher risk for low levels of blood Phe compared with children 7 years and older.
What is the mechanism of sapropterin action?
Sapropterin is a Phenylalanine Hydroxylase Activator. The mechanism of action of sapropterin is as a Phenylalanine Hydroxylase Activator, and Breast Cancer Resistance Protein Inhibitor, and P-Glycoprotein Inhibitor.
What is the function of sapropterin in nitric oxide synthesis?
Sapropterin ( tetrahydrobiopterin or BH4) is a cofactor in the synthesis of nitric oxide. It is also essential in the conversion of phenylalanine to tyrosine by the enzyme phenylalanine -4-hydroxylase; the conversion of tyrosine to L-dopa by the enzyme tyrosine hydroxylase; and conversion of tryptophan to…
How does sapropterin work in phenylalanine deficiency?
Mechanism of action of sapropterin in phenylalanine deficiency. Normal: the enzyme phenylalanine hydroxylase (PAH) converts phenylalanine from dietary intake into tyrosine which is the precursor of many neurotransmitters (e.g. dopamine). Tetrahydrobiopterin (BH4) is cofactor in this conversion with dihydrobiopterin (BH2) as product.
What is the function of sapropterin dihydrochloride?
Sapropterin dihydrochloride is a synthetic form of cofactor BH4. Excess of cofactor activates residual PAH enzyme, improving phenylalanine metabolism and decreasing phenylalanine concentrations in the blood of PKU patients.