EU/3/08/578

About

On 7 November 2008, orphan designation (EU/3/08/578) was granted by the European Commission to Orphan Europe SARL, France, for cysteamine hydrochloride for the treatment of cystinosis.

Cysteamine hydrochloride has been authorised in the EU as Cystadrops since 19 January 2017.

Key facts

Active substance
Cysteamine hydrochloride
Medicine name
Cystadrops
Disease / condition
Treatment of cystinosis
Date of first decision
07/11/2008
Outcome
Positive
EU designation number
EU/3/08/578

Review of designation

During its meeting of 6 to 8 December 2016, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/08/578 for Cystadrops (mercaptamine, previously known as cysteamine hydrochloride) as an orphan medicinal product for the treatment of cystinosis. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with cystinosis. The COMP recommended that the orphan designation of the medicine be maintained1.


1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication cannot be placed on the market.

Sponsor's contact details

Orphan Europe SARL
Immeuble Le Wilson
70 Avenue du Général de Gaulle
F-92800 Puteaux
France
Tel. +33 1 4773 6458
Fax +33 1 49 00 18 00
E-mail: info@orphan-europe.com

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

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