EU/3/11/874: Orphan designation for the treatment of Stargardt's disease

Human embryonic stem-cell-derived retinal pigment epithelial cells


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.

Key facts

Active substance
Human embryonic stem-cell-derived retinal pigment epithelial cells
Intended use
Treatment of Stargardt's disease
Orphan designation status
EU designation number
Date of designation
Astellas Pharma Europe B.V.
Sylviusweg 62
2333 BE Leiden
The Netherlands
Tel. +31 71 545 5174

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

July 2023Please note that this product was withdrawn from the Union Register of orphan medicinal products in July 2023 on request of the Sponsor.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

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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