EU/3/19/2203

Table of contents

About

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}
Disease / condition
Treatment of Netherton syndrome
Date of first decision
17/10/2019
Outcome
Positive
EU designation number
EU/3/19/2203

Sponsor's contact details

MDC RegAffairs GmbH    
Baumberger Strasse 11  
Longerich   
50737 Cologne 
Germany
Tel: +49 (0) 221 7401339
E-mail: info@lifemaxlabs.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

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