Kuvan

Описание товара

About Kuvan

Kuvan is a medicine used to treat high blood levels of phenylalanine in adults and children of all ages with the genetic disorders phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.

What it is used for

Kuvan is used to treat high blood levels of phenylalanine in patients with PKU or BH4 deficiency. These disorders prevent the body from processing the amino acid phenylalanine from dietary protein, causing it to build up in the blood to abnormally high levels, which can cause problems in the nervous system.

How it is used

Kuvan is available as soluble tablets (100 mg) or as powder (100 or 500 mg), to be dissolved in water and drunk. It can only be obtained with a prescription and treatment must be started and supervised by a doctor who has experience in treating PKU and BH4 deficiency.

How it works

The high levels of phenylalanine in the blood are due to a problem with the breakdown of phenylalanine through the enzyme ‘phenylalanine hydroxylase’. The active substance in Kuvan, sapropterin dihydrochloride, is a synthetic copy of BH4. In PKU, it works by enhancing the activity of the defective enzyme, while in BH4 deficiency it replaces the missing cofactor. These actions help restore the ability of the enzyme to convert phenylalanine into tyrosine, thereby reducing phenylalanine levels in the blood.

Active substance: Sapropterin dihydrochloride

Source: EMA EPAR