EU/3/19/2145

About

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
Risdiplam
Disease / condition
Treatment of spinal muscular atrophy
Date of first decision
26/02/2019
Outcome
Positive
EU designation number
EU/3/19/2145

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 .

Sponsor's contact details

Roche Registration GmbH
Emil-Barell-Strasse 1
79639 Grenzach-Wyhlen
Baden-Wuerttemberg
Germany
E-mail: info.orphan@roche.com

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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