EU/3/17/1965: Orphan designation for the treatment of Duchenne muscular dystrophy

metformin / l-citrulline

Table of contents


On 17 January 2018, orphan designation (EU/3/17/1965) was granted by the European Commission to Duchenne UK, United Kingdom, for metformin and L-citrulline for the treatment of Duchenne muscular dystrophy.

The sponsorship was transferred to Quality Regulatory Clinical Ireland Limited, Ireland, in July 2019.

Key facts

Active substance
  • metformin
  • l-citrulline
Intended use
Treatment of Duchenne muscular dystrophy
Orphan designation status
EU designation number
Date of designation

Quality Regulatory Clinical Ireland Limited
Priory Office Park 21
Co Dublin
A94 F660
Tel. +353 1210 0440

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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