EU/3/13/1127: Orphan designation for the treatment of B-cell acute lymphoblastic leukaemia

Inotuzumab ozogamicin


On 7 June 2013, orphan designation (EU/3/13/1127) was granted by the European Commission to Pfizer Limited, United Kingdom, for inotuzumab ozogamicin for the treatment of B-cell acute lymphoblastic leukaemia.

Inotuzumab ozogamicin has been authorised in the EU as Besponsa since 29 June 2017.

The sponsorship was transferred to Pfizer Europe MA EEIG, Belgium, in July 2018.

Key facts

Active substance
Inotuzumab ozogamicin
Intended use
Treatment of B-cell acute lymphoblastic leukaemia
Orphan designation status
EU designation number
Date of designation

Pfizer Europe MA EEIG
Boulevard de la Plaine 17
1050 Bruxelles
Brussels-Capital Region

Review of designation

During its meeting of 10 to 12 May 2017, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/13/1127 for Besponsa (inotuzumab ozogamicin), as an orphan medicinal product for the treatment of B-cell acute lymphoblastic leukaemia (ALL). 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 B-cell ALL. The COMP recommended that the orphan designation of the medicine be maintained1.

1 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: Besponsa (inotuzumab ozogamicin) for the treatment of B-cell acute lymphoblastic leukaemia (PDF/81.2 KB)

    First published: 13/07/2017
    Last updated: 13/07/2017

  • 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.

    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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