Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in February 2017 at the end of the period of market exclusivity.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.
Treatment of homocystinuria
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Documents related to this orphan designation evaluation
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: