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
Disease / condition
Treatment of Stargardt's disease
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Astellas Pharma Europe B.V.
Sylviusweg 62
2333 BE Leiden
The Netherlands
Tel. +31 71 545 5174

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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