Sapropterin dihydrochloride

Sapropterin dihydrochloride is a synthetic form of the naturally occurring cofactor tetrahydrobiopterin (BH4). It is used as a medication to treat certain inherited disorders of phenylalanine metabolism, such as phenylketonuria (PKU) and tetrahydrobiopterin deficiency.

Pharmacology

Sapropterin dihydrochloride works by increasing the activity of the enzyme phenylalanine hydroxylase (PAH). This enzyme is responsible for converting phenylalanine into tyrosine, an essential amino acid. In individuals with PKU or BH4 deficiency, PAH activity is reduced, leading to an accumulation of phenylalanine in the body. High levels of phenylalanine can be toxic to the brain and can lead to intellectual disability and other neurological problems.

Medical uses

Sapropterin dihydrochloride is used in the treatment of PKU and BH4 deficiency. It is typically used in conjunction with a phenylalanine-restricted diet to help maintain safe levels of phenylalanine in the blood.

Side effects

Common side effects of sapropterin dihydrochloride include headache, runny nose, and gastrointestinal problems such as diarrhea and vomiting. In rare cases, it can cause serious allergic reactions.

History

Sapropterin dihydrochloride was first approved for medical use in the United States in 2007. It is marketed under the brand name Kuvan by BioMarin Pharmaceutical.

See also

• Phenylketonuria
• Tetrahydrobiopterin deficiency
• BioMarin Pharmaceutical