| https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-001 |
| EU/3/00/002 | alpha-galactosidase A | 2000-08-08 | 2010-10-18 | Treatment of Fabry disease | 2011-11-18 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-002 |
| EU/3/00/003 | alpha-galactosidase A | 2000-08-08 | 2009-11-19 | Treatment of Fabry disease | 2011-11-18 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-003 |
| EU/3/00/004 | Fluorouracil | 2000-10-18 | 2009-05-15 | Treatment of glioblastoma | 2009-05-15 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-004 |
| EU/3/00/005 | gemtuzumab ozogamicin | 2000-10-18 | 2004-11-08 | Treatment of acute myeloid leukaemia | 2013-06-19 | 397c291a-151d-4cee-466e-b13fd50aa31d | Mylotarg; Mylotarg | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-005 | EMEA/H/C/004204; EMEA/H/C/000705 |
| EU/3/00/006 | 1,5-(Butylimino)-1,5-dideoxy, D-glucitol | 2000-10-18 | 2009-05-15 | Treatment of Gaucher disease | 2013-02-27 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-006 |
| EU/3/00/007 | N-carbamyl-L-glutamic acid | 2000-10-18 | 2009-03-03 | Treatment of N-acetylglutamate synthetase (NAGS) deficiency | 2014-07-25 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-007 |
| EU/3/00/008 | arsenic trioxide | 2000-10-18 | 2007-07-04 | Treatment of acute promyelocytic leukaemia | 2013-10-17 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-008 |
| EU/3/00/009 | thalidomide | 2000-12-29 | 2009-05-15 | Treatment of erythema nodosum leprosum (ENL) or type II lepra reactions | 2009-05-15 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-009 |
| EU/3/00/010 | anagrelide hydrochloride | 2000-12-30 | 2009-06-15 | Treatment of essential thrombocythaemia | 2017-02-08 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-010 |
| EU/3/00/011 | busulfan | 2000-12-30 | 2009-05-15 | Conditioning treatment prior to haematopoietic-progenitor-cell transplantation | 2013-07-31 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-011 |
| EU/3/00/012 | nitisinone | 2000-12-30 | 2009-03-03 | Treatment of tyrosinaemia type 1 | 2015-05-08 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-012 |
| EU/3/00/013 | Ethyl Eicosapentaenoate | 2000-12-29 | 2006-01-04 | Treatment of Huntington’s disease | 2006-01-04 | 3c4cc10c-eeac-c14c-72b9-479cd4fab785 | Ethyl Eicosapent soft gelatin capsules; Ethyl Eicosapent soft gelatin capsules | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-013 | EMEA/H/C/001148; EMEA/H/C/001148 |
| EU/3/00/014 | iloprost | 2000-12-30 | 2009-03-12 | Treatment of primary and of the following forms of secondary pulmonary hypertension… | 2013-10-08 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-014 |
| EU/3/00/015 | xaliproden hydrochloride | 2001-01-17 | 2009-03-11 | Treatment of amyotrophic lateral sclerosis | 2011-05-06 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-015 |
| EU/3/00/016 | Lusupultide | 2001-01-17 | 2009-05-15 | Treatment of acute respiratory distress syndrome (ARDS) | 2009-05-15 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-016 |
| EU/3/00/017 | arsenic trioxide | 2001-01-17 | 2009-05-15 | Treatment of acute promyelocytic leukaemia | 2009-05-15 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-017 |
| EU/3/01/018 | recombinant human acid alpha-glucosidase | 2001-02-15 | 2009-06-15 | Treatment of glycogen storage disease type II (Pompe's disease) | 2016-08-10 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-018 |
| EU/3/01/019 | Bosentan | 2001-02-15 | 2007-02-28 | Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | 2013-02-27 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-019 |
| EU/3/01/020 | Ibuprofen | 2001-02-15 | 2009-05-15 | Treatment of patent ductus arteriosus | 2015-03-06 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-020 |
| EU/3/01/021 | imatinib mesilate | 2001-02-15 | 2007-02-28 | Treatment of chronic myeloid leukaemia | 2015-02-10 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-021 |
| EU/3/01/022 | laronidase | 2001-02-15 | 2009-06-15 | Treatment of mucopolysaccharidosis type I | 2013-07-08 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-022 |
| EU/3/01/023 | pegvisomant | 2001-02-15 | 2005-11-29 | Treatment of acromegaly | 2013-02-27 | https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-023 |
EU Designation Number | Active Substance | Date of Designation or Refusal | First Published Date | Intended Use | Last Updated Date | Medicine | Medicine Name | Orphan Designation URL | Related EMA Product Number | Status |
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