EU/3/15/1599

About

On 14 December 2015, orphan designation (EU/3/15/1599) was granted by the European Commission to Pharma Gateway AB, Sweden, for adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B.

The sponsorship was transferred to Ultragenyx Germany GmbH in September 2020.

Key facts

Active substance
Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B-
Disease / condition
Treatment of haemophilia B
Date of first decision
14/12/2015
Outcome
Positive
EU designation number
EU/3/15/1599

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

Ultragenyx Germany GmbH
Friedrichstrasse 191
Mitte
10117 Berlin
Germany
Tel: 0041793031447
E-mail: inquiry@ultragenyx.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.

How useful was this page?

Add your rating