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
22/10/2020 Orfadin - EMEA/H/C/000555 - II/0071
- Annex I - Summary of product characteristics
- Annex IIA - Manufacturing-authorisation holder responsible for batch release
- Annex IIB - Conditions of the marketing authorisation
- Annex IIIA - Labelling
- Annex IIIB - Package leaflet
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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).