EU/3/07/474: Orphan designation for the treatment of cystic fibrosis

Alpha-1 proteinase inhibitor

Table of contents


On 14 September 2007, orphan designation (EU/3/07/474) was granted by the European Commission to CSL Behring GmbH, Germany, for alpha-1 proteinase inhibitor (inhalation use) for the treatment of cystic fibrosis.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2020 on request of the Sponsor.

Key facts

Active substance
Alpha-1 proteinase inhibitor
Intended use
Treatment of cystic fibrosis
Orphan designation status
EU designation number
Date of designation

CSL Behring GmbH
Emil-von-Behring-Str. 76
35041 Marburg
Tel. +49 6421 3912

Documents related to this orphan designation evaluation

Patients' organisations

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:

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