🇪🇺 Orfadin in European Union

EMA authorised Orfadin on 24 August 2017

Marketing authorisations

EMA — authorised 24 August 2017

  • Application: EMEA/H/C/004281
  • Marketing authorisation holder: MendeliKABS Europe Ltd
  • Local brand name: Nitisinone MDK (previously Nitisinone MendeliKABS)
  • Indication: 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.
  • Status: withdrawn

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EMA — authorised 26 July 2018

  • Application: EMEA/H/C/004582
  • Marketing authorisation holder: Cycle Pharmaceuticals (Europe) Ltd
  • Local brand name: Nityr
  • Indication: Treatment of adult and paediatric patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.
  • Status: approved

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Orfadin in other countries

Same drug, different regulators. Each page below lists the local marketing authorisation, HTA position, pricing, and adverse-event data.

Other Other approved in European Union

Frequently asked questions

Is Orfadin approved in European Union?

Yes. EMA authorised it on 24 August 2017; EMA authorised it on 26 July 2018.

Who is the marketing authorisation holder for Orfadin in European Union?

MendeliKABS Europe Ltd holds the EU marketing authorisation.