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.

Key facts

Active substance
(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  (avapritinib) 
Disease / condition
Treatment of gastrointestinal stromal tumours
Date of first decision
EU designation number

Review of designation

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

Sponsor's contact details

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

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.

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