Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in May 2012 on request of the sponsor.
On 30 April 2002, orphan designation (EU/3/02/100) was granted by the European Commission to Baxter AG, Austria, for recombinant human alpha-1 antitrypsin for the treatment of emphysema secondary to congenital alpha-1 antitrypsin deficiency.
The sponsorship was transferred to MDS Pharma Services France SAS, France, in October 2006 and subsequently to Fulcrum Pharma (Europe) Ltd., United Kingdom, in July 2008.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Recombinant human alpha-1 antitrypsin
Treatment of emphysema secondary to congenital alpha-1 antitrypsin deficiency
|Orphan designation status||
|EU designation number||
|Date of designation||
Fulcrum Pharma (Europe) Ltd
Tel. +44 (0)1442 283 600
Fax +44 (0)1442 283 613
Documents related to this orphan designation evaluation
EU/3/02/100: Public summary of positive opinion for orphan designation of recombinant human alpha-1 antitrypsin for the treatment of emphysema secondary to congenital alpha-1 antitrypsin deficiency (PDF/127.56 KB)
First published: 04/06/2012
Last updated: 13/01/2014
EMEA/COMP/681/02 Rev. 5
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
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: