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.
Treatment of spinal muscular atrophy
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Review of designation
Documents related to this orphan designation evaluation
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: