EU/3/19/2203: Orphan designation for the treatment of Netherton syndrome
(S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide}
Overview
On 17 October 2019, orphan designation EU/3/19/2203 was granted by the European Commission to MDC RegAffairs GmbH, Germany, for (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide} (also known as LM-030) for the treatment of Netherton syndrome.
Key facts
Active substance |
(S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide}
|
Intended use |
Treatment of Netherton syndrome
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/19/2203
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Date of designation |
17/10/2019
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Sponsor |
MDC RegAffairs GmbH |
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
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: