Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide
Priya Life Science Rare disease register
All regulatorsEMA approvalsEU safety letters
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 10 Nov 2026
3,310Orphan designations listed
2,201Currently active
114Designated in the last 12 months
1,073Withdrawn 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 · Synced · Editorial standards
01

Matching designations

5 matches
SubstanceIntended useStatusDesignated
treprostinil sodium
EU/3/22/2588
5 clinical trials →		 Treatment of idiopathic pulmonary fibrosis Active (positive) 16 Mar 2022
treprostinil sodium
EU/3/13/1103 · now authorised as Trepulmix
5 clinical trials →		 Treatment of chronic thromboembolic pulmonary hypertension Active (positive) 8 Feb 2013
treprostinil diethanolamine
EU/3/09/635
 Treatment of systemic sclerosis Active (positive) 15 May 2009
treprostinil diethanolamine
EU/3/05/310 · now authorised as Orepaxam
 Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension Active (positive) 26 Aug 2005
treprostinil sodium
EU/3/04/197 · now authorised as Tyvaso; Tyvaso
5 clinical trials →		 Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension Active (positive) 14 Apr 2004

New orphan designations and approvals, weekly

One email every Friday: new EMA, FDA and Health Canada approvals, EU and US medicine shortages, and the week's Irish pharma news.

Free. Unsubscribe from any email with one click. See our privacy policy.

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 10 Nov 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.