EU/3/17/1835: Orphan designation for the treatment of epidermolysis bullosa
Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (prademagene zamikeracel)
Table of contents
Overview
On 27 February 2017, orphan designation (EU/3/17/1835) was granted by the European Commission to Ser-mes Planificación SL, Spain, for ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (also known as EB-101) for the treatment of epidermolysis bullosa.
Key facts
Active substance |
Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (prademagene zamikeracel)
|
Intended use |
Treatment of epidermolysis bullosa
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/17/1835
|
Date of designation |
27/02/2017
|
Sponsor |
Pharma Gateway AB |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product it it is approved for marketing authorisation.
Update history
Date | Update |
---|---|
January 2023 | The sponsorship was transferred from Abeona Therapeutics Europe S.L., Spain to Pharma Gateway AB, Sweden. |
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: