On 20 March 2007, orphan designation (EU/3/07/440) was granted by the European Commission to The Matthews Consultancy Ltd, United Kingdom, for recombinant adeno-associated viral vector containing human alpha-1 antitrypsin gene for the treatment of congenital alpha-1 antitrypsin deficiency.
The Matthews Consultancy Ltd changed its name to TMC Pharma Services Ltd in October 2009.
The sponsorship was transferred to Diamond Roc EOOD, Bulgaria, in April 2019.
The sponsorship was transferred to Diamond Pharma Services Ireland Limited in February 2020.
Recombinant adeno-associated viral vector containing human alpha-1 antitrypsin gene
Treatment of congenital alpha-1 antitrypsin deficiency
|Orphan designation status||
|EU designation number||
|Date of designation||
Propharma Group The Netherlands B.V.
|October 2022||The sponsorship was transferred to Propharma Group The Netherlands B.V., Netherlands.|
|February 2020||The sponsorship was transferred to Diamond Pharma Services Ireland Limited.|
|April 2019||The sponsorship was transferred to Diamond Roc EOOD, Bulgaria.|
|October 2009||The Matthews Consultancy Ltd changed its name to TMC Pharma Services Ltd.|
Documents related to this orphan designation evaluation
EU/3/07/440: Public summary of positive opinion for orphan designation of recombinant adeno-associated viral vector containing human alpha-1 antitripsin gene for the treatment of congenital alpha-1 antitrypsin deficiency... (PDF/128.52 KB)
First published: 19/07/2007
Last updated: 08/04/2011
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: