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)

Table of contents

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
Groenenborgerlaan 16
2610 Antwerp
Belgium
E-mail: orphan@qbdgroup.com

Update history

DateUpdate
March 2023The sponsorship was transferred from EUDRAC GmbH, Germany to QbD Flanders, Belgium.  

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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