EU/3/12/1045: Orphan designation for the treatment of cystic fibrosis

Alpha-1 proteinase inhibitor


On 10 October 2012, orphan designation (EU/3/12/1045) was granted by the European Commission to Grifols Deutschland GmbH, Germany, for alpha-1 proteinase inhibitor (for inhalation use) for the treatment of cystic fibrosis.

Key facts

Active substance
Alpha-1 proteinase inhibitor
Intended use
Treatment of cystic fibrosis
Orphan designation status
EU designation number
Date of designation
Grifols Deutschland GmbH
Colmarer Strasse 22
D-60528 Frankfurt am Main
Tel. + +49 69 66 05 93 401

Review of designation

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

Documents related to this orphan designation evaluation

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.

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:

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