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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 · Editorial standards
01

Matching designations

2 matches
SubstanceIntended useStatusDesignated
vutrisiran
EU/3/22/2682
3 clinical trials →		 Treatment of Stargardt's disease Withdrawn 10 Aug 2022
synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and…
EU/3/18/2026 · now authorised as Amvuttra
 Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis) Active (positive) 25 May 2018

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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.

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.