EU/3/19/2168: Orphan designation for the treatment of maternally-inherited diabetes and deafness

(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidine-3-yl)chroman-2-carboxamide hydrochloride

Table of contents

Overview

On 28 June 2019, orphan designation EU/3/19/2168 was granted by the European Commission to Khondrion B.V., the Netherlands, for (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidine-3-yl)chroman-2-carboxamide hydrochloride (also known as KH176) for the treatment of maternally inherited diabetes and deafness.

Key facts

Active substance
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidine-3-yl)chroman-2-carboxamide hydrochloride
Intended use
Treatment of maternally-inherited diabetes and deafness
Orphan designation status
Positive
EU designation number
EU/3/19/2168
Date of designation
28/06/2019
Sponsor

Khondrion BV
Transistorweg 5
6534 Nijmegen
Gelderland
The Netherlands
Tel. +31 243617505
E-mail: info@khondrion.com

Update history

DateUpdate
March 2023The sponsor's address was updated.

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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