EU/3/09/716: Orphan designation for the treatment of chronic myeloid leukaemia

Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib)


On 2 February 2010, orphan designation (EU/3/09/716) was granted by the European Commission to Ariad Pharma Ltd, United Kingdom, for benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for the treatment of chronic myeloid leukaemia.

This medicine is now known as ponatinib.

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in July 2023 at the end of the 10-year period of market exclusivity.

Key facts

Active substance
Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib)
Medicine name
Intended use
Treatment of chronic myeloid leukaemia
Orphan designation status
EU designation number
Date of designation

Incyte Biosciences Distribution B.V.
Paasheuvelweg 25
1105 BP Amsterdam
The Netherlands

Review of designation

During its meeting of 16-17 April 2013, the Committee for Orphan Medicinal Products (COMP) reviewed the designations EU/3/09/715 and EU/3/09/716 for Iclusig (ponatinib1) as an orphan medicinal product for the treatment of acute lymphoblastic leukaemia and of chronic myeloid leukaemia. 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 conditions, and the existence of other methods of treatment. As other satisfactory methods of treatment for patients with these conditions 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 designations of the medicine be maintained2.

1Previously known as benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl].
2The 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.

Update history

May 2018The sponsorship was transferred to Incyte Biosciences Distribution B.V., The Netherlands.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Iclusig (ponatinib) for the treatment of acute lymphoblastic leukaemia and chronic myeloid leukaemia (PDF/132.27 KB)


    First published: 31/07/2013
    Last updated: 31/07/2013

  • 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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