Orfadin is a medicine for the treatment of: - hereditary tyrosinaemia type 1 (HT‑1) in patients of all ages who also follow diet restrictions; - alkaptonuria (AKU) in adults. These diseases occur when the body cannot fully break down certain amino acids including tyrosine. As a result, harmful substances build up, which can cause serious liver problems and liver cancer in patients with HT-1 and joint problems in patients with AKU. Orfadin contains the active substance nitisinone.
Therapeutic Indication
### Therapeutic indication Hereditary tyrosinemia type 1 (HT 1) Orfadin is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT 1) in combination with dietary restriction of tyrosine and phenylalanine. Alkaptonuria (AKU) Orfadin is indicated for the treatment of adult patients with alkaptonuria (AKU).
Therapeutic Area (MeSH)
ATC Code
A16AX04
ATC Item
nitisinone
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| nitisinone | N/A | 尼替西农 |
EMA Name
Orfadin
Medicine Name
Orfadin
Aliases
N/ANo risk management plan link.
