EU/3/17/1953: Orphan designation for the treatment of Fabry disease

Pegunigalsidase alfa

Overview

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Please note that this product (marketed as Elfabrio) was withdrawn from the Union Register of orphan medicinal products by the European Commission in March 2023 at the time of the granting of a marketing authorisation.

 

Key facts

Active substance
Pegunigalsidase alfa
Intended use
Treatment of Fabry disease
Orphan designation status
Withdrawn
EU designation number
EU/3/17/1953
Date of designation
12/12/2017
Sponsor

Chiesi Farmaceutici S.P.A.
Via Palermo 26/A
43100 Parma
Italy
Tel. +39 0521 279 793
E-mail: info@chiesigroup.com

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 PDF icon orphan designation withdrawal assessment report .

Update history

DateUpdate
May 2023Product withdrawn from the Union Register of orphan medicinal products by the European Commission at the time of the granting of a marketing authorisation.
May 2019The sponsorship was transferred to Chiesi Farmaceutici S.p.A., Italy.

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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