EU/3/00/012: Orphan designation for the treatment of tyrosinaemia type 1


Table of contents


Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in February 2015 at the end of the period of market exclusivity.

On 29 December 2000, orphan designation (EU/3/00/012) was granted by the European Commission to Swedish Orphan AB, Sweden, for nitisinone for the treatment of tyrosinaemia type 1.

Swedish Orphan AB changed name to Swedish Orphan International AB in January 2003 and then to Swedish Orphan Biovitrum International AB, in August 2013.

Nitisinone has been authorised in the EU as Orfadin since 21 February 2005.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

Key facts

Active substance
Medicine name
Intended use
Treatment of tyrosinaemia type 1
Orphan designation status
EU designation number
Date of designation
Swedish Orphan Biovitrum International AB
Drottninggatan 98
111 60 Stockholm
Tel. +46 8 4129800697 20 00
Fax +46 8 697 23 30

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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