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Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2021 on request of the Sponsor.
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec)
Treatment of Crigler-Najjar syndrome
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Review of designation
Sponsor's contact details
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.