EU/3/05/325: Orphan designation for the treatment of cystic fibrosis



On 7 November 2005, orphan designation (EU/3/05/325) was granted by the European Commission to Stricent AB, Sweden, for mannitolum for the treatment of cystic fibrosis.

Mannitolum has been authorised in the EU as Bronchitol since 13 April 2012.

Key facts

Active substance
Medicine name
Intended use
Treatment of cystic fibrosis
Orphan designation status
EU designation number
Date of designation

Pharmaxis Europe Limited
108 Q Housem, Furze Road
Dublin 18, D18AY29
Tel. +353 (0) 1431 9816

Review of designation

During its meeting of 8-9 November 2011, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/05/325 for Bronchitol (mannitol, previously known as mannitolum) as an orphan medicinal product for the treatment of cystic fibrosis. 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 satisfactory methods of treatment. As other satisfactory methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained.

Update history

April 2022This product was withdrawn from the Community Register of designated orphan medicinal products at the end of the 10-year period of market exclusivity.
January 2019The sponsorship was transferred to Pharmaxis Europe Limited, Ireland.
May 2008The sponsorship was transferred to Pharmaxis UK Limited, United Kingdom, in October 2006. The sponsor subsequently changed name to Pharmaxis Pharmaceuticals Limited.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Bronchitol (mannitol) for the treatment of cystic fibrosis (PDF/124.98 KB)


    First published: 30/10/2012
    Last updated: 30/10/2012

  • 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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