Orfadin
What Orfadin is used for
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).
Approved use, as published by the European Medicines Agency.
Key facts
- Brand
- Orfadin
- Active substance
- nitisinone
- Disease area
- Tyrosinemias
- Medicine group
- Other alimentary tract and metabolism products
- ATC code
- A16AX04
- Marketing authorisation holder
- Swedish Orphan Biovitrum International AB
- Authorised in the EU since
- 2005
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Orfadin used for?
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).
What is the active substance in Orfadin?
Orfadin contains nitisinone.
Who makes Orfadin?
The EU marketing authorisation holder is Swedish Orphan Biovitrum International AB.
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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