EU/3/14/1309: Orphan designation for the treatment of Duchenne muscular dystrophy

17α,21-Dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione (vamorolone)


On 22 August 2014, orphan designation (EU/3/14/1309) was granted by the European Commission to NDA Group AB, Sweden, for 17α,21-dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione for the treatment of Duchenne muscular dystrophy.

The medicine is now known as vamorolone.

Key facts

Active substance
17α,21-Dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione (vamorolone)
Intended use
Treatment of Duchenne muscular dystrophy
Orphan designation status
EU designation number
Date of designation

Santhera Pharmaceuticals (Deutschland) GmbH
Marie-Curie Strasse 8
D-79539 Lörrach
Tel. +49 7621 1690200
Fax +49 7621 1690201

Review of designation

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

Update history

Date Update
May 2015 The sponsorship was transferred to ReveraGen BioPharma Limited, United Kingdom
January 2019 The sponsorship was transferred to Pharma Gateway AB, Sweden
September 2022 The sponsorship was transferred from Insidereg Limited, Ireland to Pharma Gateway AB, Sweden in September 2022.

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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