EU/3/17/1884: Orphan designation for the treatment of haemophilia B

Recombinant human factor IX protein modified with three point mutations (dalcinonacog alfa)

Overview

This medicine is now known as dalcinonacog alfa.

On 20 June 2017, orphan designation (EU/3/17/1884) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for recombinant human factor IX protein modified with three point mutations (also known as CB 2679d or ISU304) for the treatment of haemophilia B.

Key facts

Active substance
Recombinant human factor IX protein modified with three point mutations (dalcinonacog alfa)
Intended use
Treatment of haemophilia B
Orphan designation status
Positive
EU designation number
EU/3/17/1884
Date of designation
20/06/2017
Sponsor

Premier Research Group S.L.
Camino Zarzuela 19
28023 Madrid
Spain
E-mail: regulatory@gccorp.com

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

DateUpdate
August 2023The sponsorship was transferred to Premier Research Group S.L., Spain in August 2023.
July 2020The sponsorship was transferred to Turnkey PharmaConsulting Ireland Limited, Ireland.
October 2019The sponsorship was transferred to Regintel Limited, Ireland. 

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:

How useful was this page?

Add your rating