EU/3/17/1889: Orphan designation for the treatment of gastrointestinal stromal tumours
(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
Table of contents
Overview
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 |
(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
|
Intended use |
Treatment of gastrointestinal stromal tumours
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/17/1889
|
Date of designation |
17/07/2017
|
Sponsor |
Blueprint Medicines (Netherlands) B.V. |
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.
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: