EU/3/14/1421: Orphan designation for the treatment of gastrointestinal stromal tumours

Ponatinib hydrochloride


On 15 January 2015, orphan designation (EU/3/14/1421) was granted by the European Commission to ARIAD Pharma Ltd, United Kingdom, for ponatinib hydrochloride for the treatment of gastrointestinal stromal tumours.

The sponsorship was transferred to Incyte Biosciences UK Ltd, United Kingdom, in November 2016.

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

Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2022 on request of the Sponsor.

Key facts

Active substance
Ponatinib hydrochloride
Intended use
Treatment of gastrointestinal stromal tumours
Orphan designation status
EU designation number
Date of designation

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

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Documents related to this orphan designation evaluation

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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