On 21 June 2011, orphan designation (EU/3/11/874) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for human embryonic stem-cell-derived retinal pigment epithelial cells for the treatment of Stargardt's disease.
The sponsorship was transferred to Astellas Pharma Europe B.V, The Netherlands, in December 2016.
Human embryonic stem-cell-derived retinal pigment epithelial cells
Treatment of Stargardt's disease
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Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
|July 2023||Please note that this product was withdrawn from the Union Register of orphan medicinal products in July 2023 on request of the Sponsor.|
Documents related to this orphan designation evaluation
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: