Sapropterin Dipharma is a medicine that is 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. Patients with these disorders cannot process the amino acid phenylalanine from dietary protein. As a result, phenylalanine builds up in the blood to abnormally high levels, causing problems in the nervous system. Sapropterin Dipharma contains the active substance sapropterin and is a ‘generic medicine’. This means that Sapropterin Dipharma contains the same active substance and works in the same way as a ‘reference medicine’ already authorised in the EU called Kuvan. For more information on generic medicines, see the question-and-answer document [here](https://www.ema.europa.eu/en/documents/medicine-qa/questions-answers-generic-medicines_en.pdf).
Therapeutic Indication
### Therapeutic indication Sapropterin Dipharma is indicated for the treatment of hyperphenylalaninaemia (HPA) in adults and paediatric patients of all ages with phenylketonuria (PKU) who have been shown to be responsive to such treatment. Sapropterin Dipharma is also indicated for the treatment of hyperphenylalaninaemia (HPA) in adults and paediatric patients of all ages with tetrahydrobiopterin (BH4) deficiency who have been shown to be responsive to such treatment.
Therapeutic Area (MeSH)
ATC Code
A16AX07
ATC Item
sapropterin
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
EMA Name
Sapropterin Dipharma
Medicine Name
Sapropterin Dipharma
Aliases
N/A