EU/3/15/1451: Orphan designation for the treatment of Alport syndrome



Please note that this product was withdrawn from the Union Register of orphan medicinal products in May 2023 on request of the Sponsor.

On 19 March 2015, orphan designation (EU/3/15/1451) was granted by the European Commission to CTI Clinical Trial and Consulting Services Europe GmbH, Germany, for 5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3' (also known as RG-012) for the treatment of Alport syndrome.

Key facts

Active substance
Intended use
Treatment of Alport syndrome
Orphan designation status
EU designation number
Date of designation

Genzyme Europe B.V.
Paasheuvelweg 25
1105 BP Amsterdam
The Nederlands
Tel. +31 2024 54000

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Update history

May 2023Product withdrawn from the Union Register of orphan medicinal products.

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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