European Union · EMA · Rare diseases

EU orphan designations

Every medicine the European Commission has designated as an orphan medicine for a rare disease, on the EMA's recommendation, with its current status.

Source: European Medicines Agency, orphan designations data (reproduced with acknowledgement of the source). Synced 11 Oct 2026

3,310Orphan designations listed

2,204Currently active

125Designated in the last 12 months

1,070Withdrawn or expired

Maintained by Sreepriya Prasannan

MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist

Compiled from EMA orphan designation records · Last verified 11 Oct 2026 · Editorial standards

Matching designations

8 matches

Substance	Intended use	Status	Designated
Eculizumab EU/3/22/2707 13 clinical trials →	Treatment of Guillain-Barré syndrome	Withdrawn	11 Oct 2022
Eculizumab EU/3/14/1304 · now authorised as Soliris 13 clinical trials →	Treatment myasthenia gravis	Active (positive)	29 Jul 2014
Eculizumab EU/3/14/1254 13 clinical trials →	Prevention of graft rejection following solid organ transplantation	Withdrawn	26 Mar 2014
Eculizumab EU/3/14/1238 13 clinical trials →	Prevention of delayed graft function after solid organ transplantation	Withdrawn	19 Feb 2014
Eculizumab EU/3/13/1185 · now authorised as Soliris 13 clinical trials →	Treatment of neuromyelitis optica spectrum disorders	Active (positive)	5 Aug 2013
Eculizumab EU/3/12/1015 13 clinical trials →	Treatment of infection-associated haemolytic uraemic syndrome	Withdrawn	4 Jul 2012
Eculizumab EU/3/09/653 13 clinical trials →	Treatment of atypical haemolytic uraemic syndrome (aHUS)	Active (positive)	24 Jul 2009
Eculizumab EU/3/03/166 13 clinical trials →	Treatment of paroxysmal nocturnal haemoglobinuria	Expired	17 Oct 2003

How this page is built

Every row is copied from the EMA's orphan designations data, reproduced with acknowledgement of the source. Each substance links to its official EMA page.

EMA orphan designations data (JSON report) ↗ active substance, intended use, EU designation number, date of designation, status and the link to each designation page

A daily job downloads the EMA's orphan designations JSON report and lists every designation, newest first. No AI drafting and no editorial pass. See our editorial standards. The the EMA data on this page was last retrieved on 11 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.