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 sponsorship was transferred to ReveraGen BioPharma Limited, United Kingdom, in May 2015.

The sponsorship was transferred to Pharma Gateway AB, Sweden, in January 2019.

Key facts

Active substance
Disease / condition
Treatment of Duchenne muscular dystrophy
Date of first decision
EU designation 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

Pharma Gateway AB
Johanneslundsvägen 2
194 61 Upplands Väsby
Stockholms Lan
Tel. +46 8 590 778 00

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.

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