EU/3/17/1889: Orphan designation for the treatment of gastrointestinal stromal tumours



On 17 July 2017, orphan designation (EU/3/17/1889) was granted by the European Commission to PhaRA bvba, Belgium, for (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine (also known as BLU-285) for the treatment of gastrointestinal stromal tumours.

This medicine is now known as avapritinib.

The sponsorship was transferred to Blueprint Medicines (Netherlands) B.V., Netherlands in May 2019.

(S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine has been authorised in the EU as Ayvakyt since 24 September 2020. 

Key facts

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

Blueprint Medicines (Netherlands) B.V.
Gustav Mahlerplein 2
1082 MA Amsterdam
The Netherlands
Tel. +31 85 888 0230

Review of designation

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

More information is available in the orphan medicine assessment report.

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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