EU/3/01/045: Orphan designation for the treatment of homocystinuria

Betaine anhydrous

Table of contents


Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in February 2017 at the end of the period of market exclusivity.

On 9 July 2001, orphan designation (EU/3/01/045) was granted by the European Commission to Orphan Europe, France, for betaine anhydrous for the treatment of homocystinuria.

Betaine anhydrous has been authorised in the EU as Cystadane since 17 February 2007.

Key facts

Active substance
Betaine anhydrous
Medicine name
Intended use
Treatment of homocystinuria
Orphan designation status
EU designation number
Date of designation
Orphan Europe
Immeuble Le Wilson
70 Avenue du Général de Gaulle
F-92800 Puteaux
Tel. +33 1 47 73 64 58
Fax +33 1 49 00 18 00

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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