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
Table of contents
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Please note that this product was withdrawn from the Union Register of orphan medicinal products in December 2022 on request of the Sponsor.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Withdrawn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 |
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
Date | Update |
---|---|
December 2022 | The product was withdrawn from the Union Register of orphan medicinal products on request of the Sponsor. |
May 2022 | The sponsor’s address was updated in May 2022. |
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: