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.
Orfadin : EPAR - Summary for the public (PDF/144.36 KB)
First published: 10/11/2009
Last updated: 11/01/2021
Orfadin : EPAR - Risk-management-plan summary (PDF/68.9 KB)
First published: 03/11/2020
Last updated: 01/03/2021
|Agency product number||
|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
|Anatomical therapeutic chemical (ATC) code||
Swedish Orphan Biovitrum International AB
|Date of issue of marketing authorisation valid throughout the European Union||
SE-112 76 Stockholm
13/04/2023 Orfadin - EMEA/H/C/000555 - IB/0082
This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.
Other alimentary tract and metabolism products
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.
Orfadin is indicated for the treatment of adult patients with alkaptonuria (AKU).