On 24 January 2013, orphan designation (EU/3/12/1098) was granted by the European Commission to Enpharma, United Kingdom, for encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor for treatment of retinitis pigmentosa.
The sponsor's address was updated in May 2018.
The sponsor’s address was updated in October 2020.
The sponsorship was transferred to Le4d Global Regulatory Science Limited, Ireland in December 2020.
The sponsor’s address was updated in April 2022.
Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor
Treatment of retinitis pigmentosa
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Review of designation
Documents related to this orphan designation evaluation
EU/3/12/1098: Public summary of opinion on orphan designation: Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor for the treatment of ret... (PDF/126.29 KB)
First published: 01/03/2013
Last updated: 01/03/2013
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: