EU/3/02/096: Orphan designation for the treatment of alkaptonuria


Table of contents


On 13 March 2002, orphan designation (EU/3/02/096) was granted by the European Commission to Swedish Orphan AB, Sweden, for nitisinone for the treatment of alkaptonuria.

In May 2014, the sponsor changed name to Swedish Orphan Biovitrum AB (publ).

Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2020 on request of the Sponsor.

Key facts

Active substance
Intended use
Treatment of alkaptonuria
Orphan designation status
EU designation number
Date of designation
Swedish Orphan Biovitrum AB (publ)
SE 112 76 Stockholm
Tel. +46 8697 2000

Documents related to this orphan designation evaluation

Patients' organisations

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

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • 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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