This medicine was designated as an orphan medicine for the treatment of mucopolysaccharidosis type I in the European Union on 15 October 2021.
This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.
Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase
Treatment of mucopolysaccharidosis type I
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|May 2022||The sponsor’s address was updated in May 2022.|
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: