On 12 December 2017, orphan designation (EU/3/17/1953) was granted by the European Commission to Protalix B.V., the Netherlands, for pegunigalsidase alfa for the treatment of Fabry disease.
Please note that this product (marketed as Elfabrio) was withdrawn from the Union Register of orphan medicinal products by the European Commission in March 2023 at the time of the granting of a marketing authorisation.
Treatment of Fabry disease
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Pegunigalsidase alfa (Elfabrio) at the time of marketing authorisation. The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the orphan designation withdrawal assessment report .
|May 2023||Product withdrawn from the Union Register of orphan medicinal products by the European Commission at the time of the granting of a marketing authorisation.|
|May 2019||The sponsorship was transferred to Chiesi Farmaceutici S.p.A., Italy.|
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: