EU/3/12/1098: Orphan designation for the treatment of retinitis pigmentosa

Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor

Overview

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.

Key facts

Active substance
Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor
Intended use
Treatment of retinitis pigmentosa
Orphan designation status
Positive
EU designation number
EU/3/12/1098
Date of designation
24/01/2013
Sponsor

Le4d Global Regulatory Science Limited
1st Floor, Penrose 1
Penrose Dock
Cork T23 KW81
Co. Cork
Ireland
Tel: +353 (87) 7460985
E-mail: zubair.le4d@hotmail.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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