This medicine is now known as volrubigene ralaparvovec.

On 22 August 2014, orphan designation (EU/3/14/1321) was granted by the European Commission to Fondazione Telethon, Italy, for adeno-associated viral vector serotype 8 containing the human UGT1A1 gene for the treatment of Crigler-Najjar syndrome.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2021 on request of the Sponsor.

Key facts

Active substance
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec)
Disease / condition
Treatment of Crigler-Najjar syndrome
Date of first decision
EU designation number

Review of designation

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

Sponsor's contact details

Fondazione Telethon
Via Varese 16
00185 Roma
Tel. +39 06 44 01 51
Fax +39 06 44 01 55 21

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.

How useful was this page?

Add your rating