EU/3/19/2145: Orphan designation for the treatment of spinal muscular atrophy



On 26 February 2019, orphan designation (EU/3/19/2145) was granted by the European Commission to Roche Registration GmbH, Germany, for risdiplam for the treatment of spinal muscular atrophy.

Risdiplam has been authorised in the EU as Evrysdi since 26 March 2021.

Key facts

Active substance
Intended use
Treatment of spinal muscular atrophy
Orphan designation status
EU designation number
Date of designation

Roche Registration GmbH
Emil-Barell-Strasse 1
79639 Grenzach-Wyhlen

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Evrysdi at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

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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