EU/3/20/2380: Orphan designation for the treatment of haemophilia A

Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII

Overview

Please note that this product was withdrawn from the Union Register of orphan medicinal products in December 2022 on request of the Sponsor.

On 9 December 2020, orphan designation EU/3/20/2380 was granted by the European Commission to TMC Pharma (EU) Limited, Ireland, for allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII (also known as SIG-001) for the treatment of haemophilia A.

 

Key facts

Active substance
Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII
Intended use
Treatment of haemophilia A
Orphan designation status
Withdrawn
EU designation number
EU/3/20/2380
Date of designation
09/12/2020
Sponsor

TMC Pharma (EU) Limited
7A Durands Court
45 Parnell Street
Waterford
Co. Waterford
X91 P381
Ireland
Tel. + 353766705745
E-mail: info@tmcpharma.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
December 2022

The product was withdrawn from the Union Register of orphan medicinal products on request of the Sponsor.

May 2022The sponsor’s address was updated in May 2022.

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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