EU/3/19/2151: Orphan designation for the treatment of haemophilia B

Marzeptacog alfa (activated)


On 1 April 2019, orphan designation (EU/3/19/2151) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for marzeptacog alfa (activated) (also known as MarzAA) for the treatment of haemophilia B.

Key facts

Active substance
Marzeptacog alfa (activated)
Intended use
Treatment of haemophilia B
Orphan designation status
EU designation number
Date of designation

Premier Research Group S.L.
Camino Zarzuela 19
28023 Madrid

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

August 2023The sponsorship was transferred to Premier Research Group S.L., Spain.
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:

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