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Community Register of medicinal products
Register of not active Orphan Medicinal Products (alphabetical)
| Product | EU Designation | Designated Orphan Indication | Sponsor | Designation date | Tradename EU Centralised Nr implemented by |
| Human monoclonal antibody against HLA-DR | EU/3/05/291 | Treatment of Hodgkin’s lymphoma | GPC Biotech AG | 16/06/2005 | |
| (-)-trans-3-(5,6-dihydro-4H-pyrrolo[3,2,1-ij]quinolin-1yl)-4-(1H-indol-3-yl) pyrrolidine-2, 5-dione | EU/3/09/678 | Treatment of soft tissue sarcoma | Gregory Fryer Associates Ltd | 8/10/2009 | |
| (1S, 2S, 3R, 4R)-3-(5-Fluoro-2-(3-methyl-4-(4-methylpiperazin-1-yl)-phenylamino)-pyrimidin-4-ylamino)-bicyclo[2.2.1]hept-5-ene-2-carboxamide benzoate | EU/3/10/729 | Treatment of acute myeloid leukaemia | Merck KGaA | 23/03/2010 | |
| (R, S)-3-(bromomethyl)-3-butanol-1-yl-diphosphate | EU/3/04/218 | Treatment of renal cell carcinoma | Innate Pharma SAS | 29/07/2004 | |
| (Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3Hindol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysuccinate | EU/3/05/268 | Treatment of renal cell carcinoma | Pfizer Ltd | 10/03/2005 | |
| (Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3Hindol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysuccinate | EU/3/05/267 | Treatment of malignant gastrointestinal stromal tumours | Pfizer Ltd | 10/03/2005 | Sutent EU/1/06/347/... 11/01/2007 |
| 17-(allylamino)-17-demethoxygeldanamycin hydroquinone hydrochloride | EU/3/07/512 | Treatment of malignant gastro intestinal stromal tumours | Voisin Consulting S.A.R.L. | 29/11/2007 | |
| 17-allylamino-17-demethoxygeldanamycin | EU/3/04/256 | Treatment of chronic myeloid leukemia | Bristol-Myers Squibb International Corporation | 26/01/2005 | |
| 17-allylamino-17-demethoxygeldanamycin | EU/3/04/247 | Treatment of multiple myeloma | Bristol-Myers Squibb International Corporation | 21/12/2004 | |
| 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine | EU/3/06/385 | Treatment of Duchenne muscular dystrophy | Vastox plc. | 25/07/2006 | |
| 2’-O-methyl-phosphorothioate oligonucleotide | EU/3/06/357 | Treatment of Duchenne muscular dystrophy | Prosensa B.V. | 16/02/2006 | |
| 26 base single stranded phosphodiester DNA oligonucleotide | EU/3/06/353 | Treatment of renal cell carcinoma | Antisoma Research Ltd | 16/02/2006 | |
| 26 base single stranded phosphodiester DNA oligonucleotide | EU/3/06/352 | Treatment of pancreatic cancer | Antisoma Research Ltd | 16/02/2006 | |
| 26 base single stranded phosphodiester DNA oligonucleotide | EU/3/09/662 | Treatment of acute myeloid leukaemia | Antisoma Research Ltd | 8/10/2009 | |
| 4,5-dihydro-2-(2,4-dihydroxyphenyl)-4-methylthiazole-4(S)-carboxylic acid | EU/3/03/180 | Treatment of chronic iron overload requiring chelation therapy | Genzyme Europe B.V. | 12/12/2003 | |
| 4-[3,5-bis(trimethylsilyl)benzamido] benzoic acid | EU/3/07/497 | Treatment of hepatocellular carcinoma | Quintiles Ireland Ltd | 22/10/2007 | |
| 5,10-methylene-tetrahydrofolic acid | EU/3/04/221 | Treatment of pancreatic cancer in combination with 5-fluorouracil | Interface International Consultancy Limited | 2/09/2004 | |
| 5-10-Methylene-tetrahydrofolate | EU/3/03/143 | Treatment of pancreatic cancer in combination with 5-fluorouracil | Interface International Consultancy Limited | 11/06/2003 | |
| 8-cyclopentlyl-1,3-dipropylxanthine | EU/3/01/030 | Treatment of cystic fibrosis | SciClone Pharmaceuticals Italy S.r.l | 29/03/2001 | |
| a -1-acid glycoprotein | EU/3/03/138 | Treatment of tricyclic antidepressants poisoning | Bio Products Laboratory | 20/03/2003 | |
| a-1-acid glycoprotein | EU/3/03/164 | Treatment of cocaine poisoning | Bio Products Laboratory | 2/10/2003 | |
| Abetimus sodium | EU/3/01/064 | Treatment of lupus nephritis | La Jolla Limited | 20/11/2001 | |
| Acetylcysteine | EU/3/04/259 | Treatment of idiopathic pulmonary fibrosis | Zambon Group Spa | 26/01/2005 | |
| 5-methyl-pyridine-2-sulfonic acid {6-(2-hydroxy-ethoxy)-5-(2-methoxy-phenoxy)-2-[2-(1H-tetrazol-5-yl)-pyridin-4-yl]-pyrimidin-4-yl}-amide sodium salt | EU/3/03/150 | Treatment of cutaneous T-cell lymphoma | Transgene S.A. | 9/07/2003 | |
| Aldesleukin (inhalation use) | EU/3/03/146 | Treatment of renal cell carcinoma | Chiron Corporation Ltd | 30/06/2003 | |
| Alfimeprase | EU/3/05/322 | Treatment of acute peripheral arterial occlusion | Quadramed Limited | 28/10/2005 | |
| Allogeneic human umbilical cord tissue-derived cells | EU/3/08/534 | Treatment of retinitis pigmentosa | Centocor, B.V. | 1/04/2008 | |
| Alpha-Galactosidase A | EU/3/00/002 | Treatment of Fabry disease | Shire Human Genetic Therapies AB | 8/08/2000 | Replagal EU/1/01/189/... 3/08/2001 |
| Alpha-Galactosidase A | EU/3/00/003 | Treatment of Fabry disease | Genzyme Europe B.V. | 8/08/2000 | Fabrazyme EU/1/01/188/... 3/08/2001 |
| Ambrisentan | EU/3/10/790 | Treatment of idiopathic pulmonary fibrosis | Gilead Sciences International Ltd | 1/10/2010 | |
| Amiloride hydrochloride dihydrate | EU/3/03/147 | Treatment of cystic fibrosis | Pulmo Tec GmbH | 30/06/2003 | |
| Amonafide L-malate | EU/3/07/483 | Treatment of acute myeloid leukaemia | Antisoma Research Limited | 22/10/2007 | |
| anti-CD147 murine monoclonal IgM | EU/3/02/123 | Treatment of Graft versus Host Disease | SangStat UK Limited | 14/11/2002 | |
| Anti-EphA2 monoclonal antibody conjugated to maleimidocaproyl monomethylauristatin phenylalanine | EU/3/09/682 | Treatment of ovarian cancer | MedImmune Ltd | 28/10/2009 | |
| Anti-von Willebrand Aptamer | EU/3/08/547 | Treatment of thrombotic thrombocytopenic purpura | FGK Representative Service GmbH | 3/06/2008 | |
| Aplidine | EU/3/03/151 | Treatment of acute lymphoblastic leukaemia | Pharma Mar SA Sociedad Unipersonal | 9/07/2003 | |
| Apomorphine (oromucosal use) | EU/3/01/072 | Treatment of off-periods in Parkinson´s disease not responding to other oral treatment | Orion Corporation | 5/12/2001 | |
| Arsenic trioxide | EU/3/00/008 | Treatment of acute promyelocytic leukaemia | Cephalon Europe | 18/10/2000 | Trisenox EU/1/02/204/... 5/03/2002 |
| Arsenic trioxide | EU/3/00/017 | Treatment of acute promyelocytic leukaemia | Agence générale des équipements et produits de santé - Etablissement pharmaceutique des hôpitaux de Paris (AGEPS-EPHP) | 17/01/2001 | |
| Arsenic trioxide | EU/3/01/031 | Treatment of myelodysplastic syndromes | Cephalon Europe | 29/03/2001 | |
| Arsenic trioxide | EU/3/07/460 | Treatment of acute myeloid leukaemia | Cephalon Europe | 2/08/2007 | |
| Arsenic trioxide | EU/3/01/032 | Treatment of multiple myeloma | Cephalon Europe | 29/03/2001 | |
| Autologous CD34+ cells transfected with retroviral vector containing the human gp91(phox) gene | EU/3/06/393 | Treatment of chronic granulomatous disease | Vision 7 GmbH | 28/08/2006 | |
| Autologous urothelial and smooth muscle cells | EU/3/08/574 | Treatment of spinal cord injury | European Medical Advisory Services Limited | 7/11/2008 | |
| Autologous urothelial and smooth muscle cells | EU/3/08/537 | Treatment of spina bifida | European Medical Advisory Services Limited | 17/03/2008 | |
| Beraprost sodium | EU/3/01/053 | Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | Aventis Pharma S.A. | 18/09/2001 | |
| Boswellia serrata resin extract | EU/3/02/117 | Treatment of peritumoral oedema derived from brain tumours | Pharmasan GmbH | 21/10/2002 | |
| Bryostatin-1 | EU/3/02/099 | Treatment of oesophageal cancer | GPC Biotech AG | 30/04/2002 | |
| Carbamic acid, [[4-[[3-[[4-[1-(4-hydroxyphenyl)-1-methyl-ethyl]phenoxy]methyl]phenyl]methoxy]-phenyl]iminomethyl]-,ethyl ester | EU/3/02/105 | Treatment of cystic fibrosis | Boehringer Ingelheim International GmbH | 26/06/2002 | |
| Carmustine (solution for intratumoral injection) | EU/3/02/085 | Treatment of glioma | Icon Clinical Research UK Ltd | 5/03/2002 | |
| Chimeric-anti-interleukin-6 monoclonal antibody | EU/3/03/148 | Treatment of renal cell carcinoma | Janssen Biologics B.V. | 30/06/2003 | |
| Ciclosporin | EU/3/04/196 | Treatment of atopic keratoconjunctivitis | Allergan Pharmaceuticals Ireland | 14/04/2004 | |
| Ciclosporin (implant) | EU/3/06/415 | Prevention of rejection of corneal transplant | Lux Biosciences GmbH | 3/11/2006 | |
| Colistimethate sodium | EU/3/02/088 | Treatment of Pseudomonas aeruginosa lung infection (including colonisation) in cystic fibrosis | Forest Laboratories UK Ltd | 19/02/2002 | |
| Deuterium oxide | EU/3/04/239 | Treatment of pancreatic cancer | BDD Berolina Drug Development GmbH | 20/10/2004 | |
| Dexamethasone (intravitreal implant) | EU/3/10/763 | Treatment of non-infectious uveitis affecting the posterior segment of the eye | Allergan Pharmaceuticals Ireland | 4/08/2010 | |
| Dihydroartemisinin, piperaquine | EU/3/07/468 | Treatment of malaria | Sigma Tau Industrie Farmaceutiche Riunite S.p.A | 3/08/2007 | |
| Doxorubicin carbon / iron magnetically targeted microparticles | EU/3/02/112 | Treatment of hepatocellular carcinoma | Interface International Consultancy Limited | 11/09/2002 | |
| Eflornithine hydrochloride | EU/3/02/087 | Treatment of Familial Adenomatous Polyposis (FAP) | ILEX Services Limited | 19/02/2002 | |
| Eltrombopag olamine | EU/3/07/467 | Treatment of idiopathic thrombocytopenic purpura | GlaxoSmithKline Trading Services Limited | 3/08/2007 | Revolade EU/1/10/612/... 11/03/2010 |
| Engineered protein inhibitor of human neutrophil elastase | EU/3/03/152 | Treatment of cystic fibrosis | Debioclinic SA | 9/07/2003 | |
| Epothilone B | EU/3/02/098 | Treatment of ovarian cancer | Novartis Europharm Ltd | 22/03/2002 | |
| Eptacog alfa (activated) | EU/3/07/436 | Treatment of post-neonatal intracerebral haemorrhage | Novo Nordisk A/S | 20/03/2007 | |
| everolimus | EU/3/07/488 | Treatment of gastro-entero-pancreatic neuroendocrine tumours | Novartis Europharm Ltd | 14/11/2007 | |
| everolimus | EU/3/07/449 | Treatment of renal cell carcinoma | Novartis Europharm Ltd | 5/06/2007 | Afinitor EU/1/09/538/... 3/08/2009 |
| Fenretinide | EU/3/06/426 | Treatment of primary malignant bone tumours | Cancer Research UK | 26/01/2007 | |
| Fenretinide | EU/3/06/427 | Treatment of soft tissue sarcoma | Cancer Research UK | 30/01/2007 | |
| Fluorouracil | EU/3/00/004 | Treatment of glioblastoma | Ethypharm S.A. | 18/10/2000 | |
| Fomepizole | EU/3/01/040 | Treatment of methanol poisoning | IDIS Ltd | 30/05/2001 | |
| GM-CSF receptor antagonist | EU/3/02/089 | Treatment of juvenile myelomonocytic leukaemia | British Biotech Pharmaceuticals Ltd | 18/03/2002 | |
| H-Tyrosine-Glycine-Phenylalanine-Glycine-Glycine-OH | EU/3/03/167 | Treatment of chronic idiopathic myelofibrosis | Abiogen Pharma S.p.A. | 20/10/2003 | |
| Human C1-inhibitor | EU/3/09/668 | Treatment of angioedema caused by C1-inhibitor deficiency | ViroPharma SPRL | 8/10/2009 | |
| Human engineered monoclonal antibody specific for Transforming Growth Factor ß1 | EU/3/01/080 | Treatment of systemic sclerosis | Genzyme Europe B.V. | 4/02/2002 | |
| Human engineered monoclonal antibody specific for Transforming Growth Factor ß2 | EU/3/01/042 | Prevention of scarring in glaucoma filtration surgical procedures | Cambridge Antibody Technology | 30/05/2001 | |
| Human Milk Fat Globule 1 / Human Milk Fat Globule 1 - S-p-isothiocyanatobenzyl-diethylenetriaminepentaacetic acid for use with 90Yttrium | EU/3/01/043 | Treatment of ovarian cancer | Antisoma Plc | 30/05/2001 | |
| Human monoclonal antibody against HLA-DR | EU/3/06/358 | Treatment of multiple myeloma | GPC Biotech AG | 28/02/2006 | |
| Human monoclonal antibody against HLA-DR | EU/3/05/347 | Treatment of chronic lymphocytic leukaemia | GPC Biotech AG | 24/01/2006 | |
| Human monoclonal hepatitis B immunoglobulins | EU/3/04/187 | Prevention of hepatitis B re-infection following liver transplantation | ICON Clinical Research (UK) Ltd | 23/02/2004 | |
| Human Staphylococcus aureus polyclonal immunoglobulin and human Staphylococcus epidermidis polyclonal immunoglobulin | EU/3/05/335 | Prevention of late onset sepsis in premature infants of less or equal than 32 weeks gestational age | Omnicare Clinical Research | 14/12/2005 | |
| Human transferrin conjugated to mutant diphtheria toxin | EU/3/02/090 | Treatment of gliomas | Xenova Biomedix Limited | 19/03/2002 | |
| Humanised Anti-HM1.24 Monoclonal Antibody | EU/3/01/037 | Treatment of multiple myeloma | Chugai Pharma Europe Ltd | 10/05/2001 | |
| Humanized anti-KSA monoclonal antibody - human interleukin-2 fusion protein | EU/3/02/097 | Treatment of renal cell carcinoma | Merck KGaA | 22/03/2002 | |
| H-Val-Ile-Val-Lys-Leu-Ile-Pro-Ser-Thr-Ser-Ser-Ala-Val-Asp-Thr-Pro-Tyr-Leu-Asp-Ile-Thr-Tyr-His-Phe-Val-Ala-Gln-Arg-Leu-Pro-Leu-OH | EU/3/06/398 | Treatment of myasthenia gravis | Debioclinic SA | 28/08/2006 | |
| Ibuprofen | EU/3/01/027 | Prevention of patent ductus arteriosus in premature neonates of less than 34 weeks of gestational age | Orphan Europe S.a.r.l. | 5/03/2001 | |
| Imatinib mesilate | EU/3/05/304 | Treatment of acute lymphoblastic leukaemia | Novartis Europharm Ltd | 26/08/2005 | Glivec EU/1/01/198/... 13/09/2006 |
| Imatinib mesilate | EU/3/05/305 | Treatment of dermatofibrosarcoma protuberans | Novartis Europharm Ltd | 26/08/2005 | Glivec EU/1/01/198/... 13/09/2006 |
| Imatinib | EU/3/01/061 | Treatment of malignant gastrointestinal stromal tumours | Novartis Europharm Limited | 20/11/2001 | Glivec EU/1/01/198/... 24/05/2002 |
| Imatinib mesilate | EU/3/05/340 | Treatment of myelodysplastic / myeloproliferative diseases | Novartis Europharm Ltd | 23/12/2005 | Glivec EU/1/01/198/... 28/11/2006 |
| Imatinib mesilate | EU/3/05/306 | Treatment of mastocytosis | Novartis Europharm Ltd | 26/08/2005 | |
| Imatinib mesilate | EU/3/05/320 | Treatment of chronic eosinophilic leukaemia and the hypereosinophilic syndrome | Novartis Europharm Ltd | 28/10/2005 | Glivec EU/1/01/198/... 28/11/2006 |
| Imatinib mesylate | EU/3/01/021 | Treatment of chronic myeloid leukaemia | Novartis Europharm Ltd | 14/02/2001 | Glivec EU/1/01/198/... 7/11/2001 |
| Interferon gamma | EU/3/05/281 | Treatment of idiopathic pulmonary fibrosis | InterMune Europe Ltd | 27/05/2005 | |
| Iodine (131I) anti-CEA sheep-human chimeric monoclonal antibody monoclonal antibody labeled with iodine-131 | EU/3/03/142 | Treatment of pancreatic cancer | Xenova Biomedix Limited | 7/05/2003 | |
| Lintuzumab | EU/3/09/627 | Treatment of acute myeloid leukaemia | Seattle Genetics UK Limited | 30/04/2009 | |
| Lintuzumab | EU/3/09/626 | Treatment of myelodysplastic syndromes | Seattle Genetics UK Limited | 29/04/2009 | |
| Lumiliximab | EU/3/08/528 | Treatment of chronic lymphocytic leukaemia | Biogen Idec Limited | 4/02/2008 | |
| Lusupultide | EU/3/00/016 | Treatment of acute respiratory distress syndrome | Nycomed GmbH | 17/01/2001 | |
| Lusupultide | EU/3/07/457 | Treatment of aspiration pneumonitis requiring intubation and mechanical ventilation | Nycomed GmbH | 10/07/2007 | |
| Mecasermin rinfabate | EU/3/06/378 | Treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects | Insmed Europe Ltd. | 20/06/2006 | |
| Mecasermin rinfabate | EU/3/06/377 | Treatment of patients with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH | Insmed Europe Ltd. | 20/06/2006 | |
| Mitotane | EU/3/02/113 | Treatment of adrenal cortical carcinoma | Agence générale des équipements et produits de santé - Etablissement pharmaceutique des hôpitaux de Paris (AGEPS-EPHP) | 11/09/2002 | |
| Monoclonal antibody to human interleukin-6 | EU/3/02/125 | Treatment of post-transplant lymphoproliferative disorders | EUSA Pharma SAS | 18/12/2002 | |
| Murine monoclonal antibody to GD2 | EU/3/09/644 | Treatment of neuroblastoma | United Therapeutics Europe Ltd | 12/06/2009 | |
| Myristoylated-peptidyl-recombinant Human CD59 | EU/3/02/114 | Treatment of paroxysmal nocturnal haemoglubinuria | Orexo AB | 11/09/2002 | |
| Myristoylated-peptidyl-recombinant SCR1-3 of human complement receptor type I | EU/3/02/108 | Prevention of post transplantation graft dysfunction | Orexo AB | 30/07/2002 | |
| N-acetylsarcosyl-glycyl-L-valyl-D-allo-isoleucyl-L-threonyl-L-norvalyl-L-isoleucyl-L-arginyl-L-prolyl-N-ethylamide | EU/3/03/181 | Treatment of soft tissue sarcoma | Abbott International European Office | 12/12/2003 | |
| Nolatrexed | EU/3/03/165 | Treatment of hepatocellular carcinoma | Scirex Ltd. | 2/10/2003 | |
| Octovalent Pseudomonas aeruginosa O-polysaccharie-toxin A conjugate vaccine | EU/3/01/076 | Prevention of Pseudomonas aeruginosa infections in patients with cystic fibrosis | Orphan Europe S.a.r.l. | 11/12/2001 | |
| Opebacan | EU/3/06/402 | Treatment of meningococcal disease | XOMA Ireland Ltd | 28/08/2006 | |
| Pagibaximab | EU/3/10/743 | Prevention of sepsis caused by Gram positive pathogens in premature infants less than or equal to 34 weeks of gestational age | Theorem Clinical Research GmbH | 9/06/2010 | |
| Panobinostat | EU/3/09/721 | Treatment of Hodgkin's lymphoma | Novartis Europharm Ltd | 2/02/2010 | |
| Panobinostat lactate | EU/3/07/464 | Treatment of cutaneous T-cell lymphoma | Novartis Europharm Ltd | 2/08/2007 | |
| Paromomycin sulphate | EU/3/05/271 | Treatment of visceral leishmaniasis | Sestria Ltd | 11/04/2005 | |
| Patupilone | EU/3/09/688 | Treatment of fallopian tube cancer | Novartis Europharm Ltd | 5/11/2009 | |
| Patupilone | EU/3/09/687 | Treatment of primary peritoneal cancer | Novartis Europharm Ltd | 5/11/2009 | |
| Pazopanib hydrochloride | EU/3/06/382 | Treatment of renal cell carcinoma | Glaxo Group Limited | 29/06/2006 | |
| Pemetrexed disodium | EU/3/01/060 | Treatment of malignant mesothelioma | Eli Lilly Nederland BV | 17/09/2001 | |
| Pixantrone dimaleate | EU/3/09/722 | Treatment of diffuse large B-cell lymphoma | CTI Life Sciences Ltd | 2/02/2010 | |
| Plitidepsin | EU/3/10/838 | Treatment of post-essential thrombocythaemia myelofibrosis | Pharma Mar S.A. Sociedad Unipersonal | 23/02/2011 | |
| Plitidepsin | EU/3/10/837 | Treatment of primary myelofibrosis | Pharma Mar S.A. Sociedad Unipersonal | 23/02/2011 | |
| Plitidepsin | EU/3/10/839 | Treatment of post-polycythaemia vera myelofibrosis | Pharma Mar S.A. Sociedad Unipersonal | 23/02/2011 | |
| Porcine lung surfactant | EU/3/01/057 | Treatment of acute lung injury | Leo Pharmaceutical Products | 19/09/2001 | |
| Purified inactivated Japanese encephalitis SA14-4-2 virus vaccine | EU/3/05/348 | Prevention of Japanese encephalitis | Intercell AG | 24/01/2006 | Ixiaro EU/1/08/501/... 31/03/2009 |
| Ranpirnase | EU/3/01/033 | Treatment of malignant mesothelioma | MoRa Pharma GmbH | 29/03/2001 | |
| Recombinant adeno-associated viral vector containing human acid alpha-glucosidase-gene | EU/3/07/454 | Treatment of glycogen storage disease type II (Pompe's disease) | TMC Pharma Services Ltd. | 9/07/2007 | |
| Recombinant human alpha-1 antitrypsin | EU/3/02/100 | Treatment of emphysema secondary to congenital alpha-1-antitrypsin deficiency | Aptiv Solutions (UK) Ltd | 30/04/2002 | |
| Recombinant human alpha-1-antitrypsin (respiratory use) | EU/3/01/041 | Treatment of emphysema secondary to congenital alpha 1-antitrypsin deficiency | Bayer HealthCare AG | 30/05/2001 | |
| Recombinant human arylsulfatase A | EU/3/03/158 | Treatment of metachromatic leukodystrophy | Shire Pharmaceuticals Ireland Limited | 9/07/2003 | |
| Recombinant human C1-inhibitor | EU/3/01/036 | Treatment of angioedema caused by C1 inhibitor deficiency | Pharming Group N.V. | 11/05/2001 | |
| Recombinant human insulin-like growth factor-I / recombinant human insulin-like growth factor binding protein-3 | EU/3/04/237 | Treatment of Rabson-Mendenhall syndrome | Dr Geoffrey Allan | 21/10/2004 | |
| Recombinant human insulin-like growth factor-I / recombinant human insulin-like growth factor binding protein-3 | EU/3/04/238 | Treatment of Leprechaunism | Insmed Europe Ltd | 21/10/2004 | |
| Recombinant human insulin-like growth factor-I / recombinant human insulin-like growth factor binding protein-3 | EU/3/04/236 | Treatment of Type A extreme insulin resistance syndrome | Dr Geoffrey Allan | 21/10/2004 | |
| Recombinant human insulin-like growth factor-I / recombinant human insulin-like growth factor binding protein-3 | EU/3/04/235 | Treatment of Type B extreme insulin resistance syndrome | Insmed Europe Ltd | 20/10/2004 | |
| Recombinant human insulin-like growth factor-I / recombinant human insulin-like growth factor binding protein-3 | EU/3/03/159 | Treatment of primary growth hormone insensitivity syndrome (Laron Syndrome) | Insmed Europe Ltd | 9/07/2003 | |
| Recombinant human monoclonal antibody to hsp90 | EU/3/01/073 | Treatment of invasive fungal infections | Novartis Europharm Ltd | 5/12/2001 | |
| Recombinant human monoclonal antibody to human IL-1beta of the IgG1/K class | EU/3/08/527 | Treatment of systemic-onset juvenile idiopathic arthritis | Novartis Europharm Ltd | 4/02/2008 | |
| Recombinant human monoclonal antibody to human IL-1beta of the IgG1/K class | EU/3/07/439 | Treatment of cryopirin-associated periodic syndromes (Familial Cold Urticaria Syndrome (FCUS), Muckle-Wells Syndrome (MWS), and Neonatal Onset Multisystem Inflammatory Disease (NOMID), also known as Chronic Infantile Neurological Cutaneous | Novartis Europharm Ltd | 20/03/2007 | Ilaris EU/1/09/564/... 23/10/2009 |
| Recombinant human residue 41 glutamic acid to glutamine variant of Interferon-alfa-2b | EU/3/08/606 | Treatment of Behçet's disease | Creabilis Therapeutics S.p.A. | 19/01/2009 | |
| Ribavirin | EU/3/01/024 | Treatment of haemorrhagic fever with renal syndrome | ICN Pharmaceuticals Limited | 14/02/2001 | |
| Ribavirin | EU/3/01/029 | Treatment of adenovirus infection in immunocompromised patients | Valeant Pharmaceuticals Limited | 8/03/2001 | |
| Sabarubicin | EU/3/04/255 | Treatment of small cell lung cancer | Menarini Ricerche S.p.A. | 21/12/2004 | |
| Sapropterin | EU/3/05/308 | Treatment of hyperphenylalaninemia | Dr Gunter Schaub | 26/08/2005 | |
| Seocalcitol | EU/3/01/052 | Treatment of hepatocellular carcinoma | Leo Pharmaceutical Products | 31/07/2001 | |
| Sinapultide, dipalmitoylfosfatidylcholine, palmitoyloleoyl fosfatidylglycerol and palmitic acid | EU/3/01/054 | Treatment of Meconium Aspiration Syndrome | Discovery Laboratories, Inc | 19/09/2001 | |
| Sodium dichloroacetate | EU/3/04/225 | Treatment of systemic monochloroacetate poisoning | Dr Anthony W. Fox | 2/09/2004 | |
| Sodium oxybate | EU/3/02/131 | Treatment of narcolepsy | UCB Pharma Ltd. | 3/02/2003 | Xyrem EU/1/05/312/... 13/10/2005 |
| Sodium valproate | EU/3/05/309 | Treatement of 5q muscular atrophy | The Jennifer Trust for Spinal Muscular Atrophy | 26/08/2005 | |
| Streptococcus pyogenes Su strain cells treated with benzylpenicillin | EU/3/09/714 | Treatment of congenital lymphatic malformations | Theradex (Europe) Ltd. | 29/01/2010 | |
| Sulfonated monophosphorylated mannose oligosaccharide | EU/3/07/482 | Treatment of hepatocellular carcinoma | Constella Group Ltd | 14/09/2007 | |
| Tacrolimus hydrate | EU/3/03/185 | Treatment of vernal keratoconjunctivitis | Sucampo Pharma Europa Ltd | 14/01/2004 | |
| Talampanel | EU/3/09/631 | Treatment of glioma | Teva Pharma GmbH | 29/04/2009 | |
| Talampanel | EU/3/09/646 | Treatment of amyotrophic lateral sclerosis | Teva Pharma GmbH | 12/06/2009 | |
| Tegafur / gimeracil / oteracil | EU/3/07/515 | Treatment of gastric cancer | Taiho Pharma Europe Limited | 20/12/2007 | |
| Thalidomide | EU/3/01/066 | Treatment of erythema nodosum leprosum (ENL) or type II lepra reactions | Celgene Europe Limited | 20/11/2001 | |
| Thalidomide | EU/3/01/068 | Treatment of multiple myeloma | Kendle International Ltd | 19/12/2001 | |
| Thalidomide | EU/3/01/046 | Treatment of Graft versus Host Disease | Pharmion France | 9/07/2001 | |
| Thalidomide | EU/3/01/047 | Treatment of multiple myeloma | Pharmion France | 9/07/2001 | |
| Thalidomide | EU/3/00/009 | Treatment of erythema nodosum leprosum (ENL) or type II lepra reactions | Pharmion France | 29/12/2000 | |
| Tilarginine acetate | EU/3/05/330 | Treatment of cardiogenic shock | Dr Ulrich Granzer | 18/10/2005 | |
| Troxacitabine | EU/3/05/311 | Treatment of acute myeloid leukaemia | Pharm Research Associates (UK) Limited | 26/08/2005 | |
| Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) | EU/3/04/254 | Treatment of myelodysplastic syndromes | Accelsiors CRO & Consultancy Services GmbH | 21/12/2004 | |
| Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) | EU/3/04/252 | Treatment of chronic myeloid leukemia | Accelsiors CRO & Consultancy Services GmbH | 21/12/2004 | |
| Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) | EU/3/04/253 | Treatment of acute myeloid leukaemia | Accelsiors CRO & Consultancy Services GmbH | 21/12/2004 | |
| vandetanib | EU/3/05/344 | Treatment of medullary thyroid carcinoma | AstraZeneca UK Limited | 24/01/2006 | |
| Xaliproden hydrochloride | EU/3/00/015 | Treatment of amyotrophic lateral sclerosis | SANOFI | 17/01/2001 |