EU/3/14/1264: Orphan designation for the treatment of lymphoplasmacytic lymphoma



On 29 April 2014, orphan designation (EU/3/14/1264) was granted by the European Commission to Janssen-Cilag International N.V., Belgium, for ibrutinib for the treatment of lymphoplasmacytic lymphoma.

Ibrutinib for treatment of lymphoplasmacytic lymphoma has been authorised in the EU as Imbruvica since 21 October 2015.

Key facts

Active substance
Medicine name
Intented use
Treatment of lymphoplasmacytic lymphoma
Orphan designation status
EU designation number
Date of designation

Janssen-Cilag International N.V.
Turnhoutseweg, 30
2340 Beerse
Tel. +32 146 031 89
Fax +32 146 055 33

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Imbruvica at the time of change to the terms of the marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

During its meeting of 16 to 18 June 2015, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/14/1264 for Imbruvica (ibrutinib) as an orphan medicinal product for the treatment of lymphoplasmacytic lymphoma (also known as Waldenström's macroglobulinaemia). The COMP assessed whether, at the time of addition of a new indication to the marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness 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 lymphoplasmacytic lymphoma. 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 a comparable 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 addition of a new indication to the marketing authorisation: Imbruvica (ibrutinib) for the treatment of lymphoplasmacytic lymphoma (PDF/80.13 KB)


    First published: 08/09/2015
    Last updated: 14/09/2015

  • 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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