On 20 March 2013, orphan designation (EU/3/13/1114) was granted by the European Commission to Polyphor UK, United Kingdom, for cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt for the treatment of congenital alpha-1 antitrypsin deficiency.

The sponsorship was transferred to Santhera Pharmaceuticals (Deutschland) GmbH, Germany in June 2018.

Key facts

Active substance
Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt
Disease / condition
Treatment of congenital alpha-1 antitrypsin deficiency
Date of decision
Orphan decision number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Santhera Pharmaceuticals (Deutschland) GmbH
Marie-Curie Strasse 8
D-79539 Lörrach
Tel. +49 7621 1690200

Patients' organisations

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.

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