EU/3/20/2363: Orphan designation for the treatment of primary ciliary dyskinesia
3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, Sodium chloride solution 4.2% (w/v)
Overview
On 16 November 2020, orphan designation EU/3/20/2363 was granted by the European Commission to EUDRAC GmbH, Germany, for 3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, sodium chloride solution 4.2% (w/v) (also known as P-1037 Inhalation Solution) for the treatment of primary ciliary dyskinesia.
Key facts
Active substance |
3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, Sodium chloride solution 4.2% (w/v)
|
Intended use |
Treatment of primary ciliary dyskinesia
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/20/2363
|
Date of designation |
16/11/2020
|
Sponsor |
QbD Flanders |
Update history
Date | Update |
---|---|
March 2023 | The sponsorship was transferred from EUDRAC GmbH, Germany to QbD Flanders, Belgium. |
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: