<p>Orfadin is a medicine for the treatment of:</p><ul><li>hereditary tyrosinaemia type 1 (HT‑1) in patients of all ages who also follow diet restrictions;</li><li>alkaptonuria (AKU) in adults.</li></ul><p>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.</p><p>Orfadin contains the active substance nitisinone.</p>
Therapeutic Indication
<div class="subsection mt-3-5"> <h3>Therapeutic indication</h3><p><u>Hereditary tyrosinemia type 1 (HT 1)</u><br>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.</p> <p><u>Alkaptonuria (AKU)</u><br>Orfadin is indicated for the treatment of adult patients with alkaptonuria (AKU).</p></div>
Therapeutic Area (MeSH)
ATC Code
A16AX04
ATC Item
N/A
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| nitisinone | N/A | nitisinone |
EMA Name
Orfadin
Medicine Name
Orfadin
Aliases
N/ANo risk management plan link.
