EU/3/12/963: Orphan designation for the treatment of cutaneous T-cell lymphoma



On 22 May 2012, orphan designation (EU/3/12/963) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for chlormethine for the treatment of cutaneous T-cell lymphoma.

The sponsorship was transferred to Actelion Registration Limited, United Kingdom, in April 2014.

Chlormethine has been authorised in the EU as Ledaga since 3 March 2017.

The sponsorship was transferred to Helsinn Birex Pharmaceuticals Ltd, Ireland, in December 2018.

Key facts

Active substance
Intended use
Treatment of cutaneous T-cell lymphoma
Orphan designation status
EU designation number
Date of designation

Helsinn Birex Pharmaceuticals Ltd Damastown
Dublin 15
Tel:  +353 1 808 96 46

Review of designation

During its meeting of 17 to 19 January, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/12/963 for Ledaga (chlormethine) as an orphan medicinal product for the treatment of cutaneous T-cell lymphoma. 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 cutaneous T-cell lymphoma. The COMP recommended that the orphan designation of the medicine be maintained*.

The 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.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation Ledaga (chlormethine) for the treatment of cutaneous T-cell lymphoma (PDF/80.8 KB)

    First published: 16/03/2017
    Last updated: 16/03/2017

  • 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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