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

Marzeptacog alfa (activated)

Overview

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.

The sponsorship was transferred to Regintel Limited, Ireland, in October 2019.

The sponsorship was transferred to Turnkey PharmaConsulting Ireland Limited, Ireland, in July 2020.

Key facts

Active substance
Marzeptacog alfa (activated)
Intended use
Treatment of haemophilia B
Orphan designation status
Positive
EU designation number
EU/3/19/2151
Date of designation
01/04/2019
Sponsor

Turnkey PharmaConsulting Ireland Limited
Ellerman House 
Cratloe Wood 
Cratloe V95 X925
County Clare 
Ireland 
Tel: +353 86 844 6816 
E-mail: markturner@turnkeypharmaconsulting.com

Review of designation

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

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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