List of marketing authorised Orphan Drugs in Europe January 2008 Orphan drugs by tradename in alphabetical order Orphan drugs by decreasing of marketing authorisation Orphan drugs in therapeutic areas authorisation holder Orphanet Rep rts Series Method This document provides a list of all orphan drugs that have received an European marketing authorisation for the stated in the title. These medicinal products may now be accessible in some European countries, though not necessarily all, as in many countries additional administrative clearance by the country s authorities (for example, setting of price and public health insurance reimbursement tariffs) is required. This list is determined by cross-referencing the list of medicinal products that have been granted an orphan earlier in development with the list of medicinal products which have been granted marketing authorisation. Both lists are accessible on the DG Enterprise website (Directorate General) of the European Commission. The list of orphan drugs is arranged by: 1- tradename 2- of marketing authorisation 3- therapeutic area 4- marketing authorisation holder The document gives information on the tradename, the active substance, the designated orphan, the marketing authorisation holder and. You may find additional information on each product on the EMEA website (European Medicines Agency). The EMEA listing covers all drugs, not just orphan drugs. Information is available in up to 21 European languages. For any questions or comments, please contact us: orphanet@orpha.net
Orphan drugs by tradename in alphabetical order ALDURAZYME Laronidase Treatment of Mucopolysaccharidosis, type I ATRIANCE Nelarabine BUSILVEX CARBAGLU Busulfan (Intravenous use) N-carbamyl-Lglutamic acid INN=carglumic acid Conditioning treatment prior to hematopoietic progenitor cell transplantation Treatment of N-acetylglutamate synthetase (NAGS) deficiency Genzyme B.V. 10/06/2003 GlaxoSmithKline Research & Development Pierre Fabre Médicament 22/08/2007 09/07/2003 Orphan Europe 24/01/2003 CYSTADANE Betaine anhydrous Treatment of homocystinuria Orphan Europe 15/02/2007 DIACOMIT Stiripentol Treatment of severe myoclonic Biocodex 04/01/2007 epilepsy in infancy ELAPRASE Idursulfase Treatment of Mucopolysaccharidosis, TKT UK Ltd 08/01/2007 type II (Hunter Syndrome) EVOLTRA Clofarabine Bioenvision 29/05/2006 EXJADE Deferasirox Chronic iron overload requiring chelation therapy FABRAZYME Alpha-Galactosidase A Treatment of Fabry disease INN=agalsidase beta GLIOLAN 5-aminolevulinic acid hydrochloride Intra-operative photodynamic diagnosis of residual glioma GLIVEC Imatinib mesilate Treatment of chronic myeloid Treatment of malignant gastrointestinal Treatment of dermatofibrosarcoma protuberans Treatment of chronic eosinophilic and the hypereosinophilic Treatment of myelodysplastic / myeloproliferative diseases INCRELEX Mecasermin Treatment of primary insulinlike growth factor-1 deficiency due to molecular or genetic defects INOVELON Rufinamide Treatment of Lennox-Gastaut Genzyme Europe B.V. Medac Gesellschaft für klinische Spezialpräparate mbh Tercica Europe 28/08/2006 03/08/2001 07/09/2007 07/11/2001 03/08/2007 Eisai 16/01/2007
LITAK Cladribine (subcutaneous use) Treatment of indolent non- Hodgkin s lymphoma LYSODREN Mitotane Treatment of adrenal cortical MYOZYME NAGLAZYME Recombinant human acid alpha-glucosidase INN=alglucosidase alfa N-acetylgalactosamine-4- sulfatase INN=galsulfase Treatment of Glycogen Storage Disease type II (Pompe s disease) Treatment of Mucopolysaccharidosis, type VI (Maroteaux- Lamy Syndrome) NEXAVAR Sorafenib Treatment of renal cell Treatment of hepatocellular ONSENAL Celecoxib Treatment of Familial Adenomatous Polyposis ORFADIN Nitisinone Treatment of tyrosinaemia type I PEDEA Ibuprofen Treatment of a haemodynamically significant patent ductus arteriosus in preterm newborn infants less than 34 weeks of gestational age PHOTOBARR PRIALT REPLAGAL REVATIO Porfimer sodium (for use with photodynamic therapy) Ziconotide (intraspinal use) Alpha-Galactosidase A INN=agalsidase alfa Sildenafil citrate INN=sildenafil Treatment of high-grade dysplasia in Barrett s Esophagus Treatment of chronic pain requiring intraspinal analgesia Lipomed GmbH 14/04/2004 Laboratoire HRA Pharma Genzyme Europe B.V. BioMarin Europe Ltd Bayer Healthcare AG 28/04/2004 29/03/2006 24/01/2006 19/07/2006 Pharmacia-Pfizer 17/10/2006 EEIG Swedish Orphan 21/02/2005 International AB Orphan Europe 29/07/2004 Axcan Pharma International BV Elan Pharma International Ltd. 25/03/2004 21/02/2005 Treatment of Fabry disease TKT UK Ltd. 03/08/2001 and chronic REVLIMID Lenalidomide Treatment of Multiple Myeloma SAVENE Dexrazoxane Treatment of anthracycline extravasations SIKLOS Hydroxycarbamide Treatment of sickle cell SOLIRIS Eculizumab Treatment of paroxysmal nocturnal haemoglobinuria Pfizer 28/10/2005 Celgene Europe 14/06/2007 TopoTarget A/S 28/07/2006 OTL Pharma 29/06/2007 Alexion Europe SAS 20/06/2007 SOMAVERT Pegvisomant Treatment of acromegaly Pfizer 13/11/2002 SPRYCEL Dasatinib Treatment of chronic myeloid Bristol-Myers 20/11/2006 Squibb Pharma EEIG
SUTENT Sunitinib malate INN=sunitinib Treatment of malignant gastrointestinal Treatment of renal cell TASIGNA Nilotinib Treatment of chronic myeloid THELIN Sitaxentan sodium and chronic TORISEL Temsirolimus Treatment of renal cell TRACLEER Bosentan monohydrate INN=bosentan and chronic Treatment of Systemic Sclerosis TRISENOX Arsenic Trioxide Treatment of acute promyelocytic VENTAVIS Iloprost Treatment of primary and of the following forms of secondary pulmonary : connective tissue disease pulmonary, drug-induced pulmonary, portopulmonary, pulmonary associated with congenital heart disease, chronic WILZIN Zinc acetate dihydrate XAGRID Anagrelide Hydrochloride INN=anagrelide Pfizer 19/07/2006 Encysive (UK) Wyeth Europa Actelion Registration 19/11/2007 10/08/2006 19/11/2007 15/05/2002 Cell Therapeutics 05/03/2002 (UK) Schering AG 16/09/2003 Treatment of Wilson s disease Orphan Europe 13/10/2004 Treatment of essential thrombocythaemia Shire Pharmaceutical Development Ltd 16/11/2004 XYREM Sodium oxybate Treatment of narcolepsy UCB Pharma Ltd. 13/10/2004 YONDELIS Trabectedin Treatment of soft tissue Pharma Mar SA 17/09/2007 sarcoma ZAVESCA Miglustat Treatment of Gaucher Disease Actelion Registration 20/11/2002 INN : International Nonproprietary Name
Orphan drugs by decreasing of marketing authorisation Date (dd/mm/yyyy) 19/11/2007 TASIGNA Nilotinib Treatment of chronic myeloid 19/11/2007 TORISEL Temsirolimus Treatment of renal cell 17/09/2007 YONDELIS Trabectedin Treatment of soft tissue sarcoma 07/09/2007 GLIOLAN 5-aminolevulinic acid hydrochloride Intra-operative photodynamic diagnosis of residual glioma 22/08/2007 ATRIANCE Nelarabine 03/08/2007 INCRELEX Mecasermin Treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects 29/06/2007 SIKLOS Hydroxycarbamide Treatment of sickle cell 20/06/2007 SOLIRIS Eculizumab Treatment of paroxysmal nocturnal haemoglobinuria 14/06/2007 REVLIMID Lenalidomide Treatment of Multiple Myeloma 15/02/2007 CYSTADANE Betaine anhydrous Treatment of homocystinuria 16/01/2007 INOVELON Rufinamide Treatment of Lennox-Gastaut 08/01/2007 ELAPRASE Idursulfase Treatment of Mucopolysaccharidosis, type II (Hunter Syndrome) 04/01/2007 DIACOMIT Stiripentol Treatment of severe myoclonic epilepsy in infancy 20/11/2006 SPRYCEL Dasatinib Treatment of chronic myeloid 28/08/2006 EXJADE Deferasirox Chronic iron overload requiring chelation therapy 10/08/2006 THELIN Sitaxentan sodium and chronic 28/07/2006 SAVENE Dexrazoxane Treatment of anthracycline extravasations 19/07/2006 NEXAVAR Sorafenib Treatment of renal cell Treatment of hepatocellular holder Wyeth Europa Pharma Mar SA Medac GmbH Glaxo Group Tercica Europe Addmedica SAS Alexion Europe SAS Celgene Europe Orphan Europe Eisai Shire Human Genetic Therapies AB Biocodex Bristol-Myers Squibb Pharma EEIG Encysive (UK) TopoTarget A/S Bayer Healthcare AG
Date (dd/mm/yyyy) 19/07/2006 SUTENT Sunitinib malate INN=sunitinib Treatment of malignant gastrointestinal Treatment of renal cell 29/05/2006 EVOLTRA Clofarabine 29/03/2006 MYOZYME Recombinant human acid alpha-glucosidase INN=alglucosidase alfa 24/01/2006 NAGLAZYME N-acetylgalactosamine-4-sulfatase INN=galsulfase 28/10/2005 REVATIO Sildenafil citrate INN=sildenafil Treatment of Glycogen Storage Disease type II (Pompe s disease) Treatment of Mucopolysaccharidosis, type VI (Maroteaux- Lamy Syndrome) and chronic holder Pfizer Bioenvision Genzyme Europe B.V. BioMarin Europe Ltd Pfizer 13/10/2004 XYREM Sodium oxybate Treatment of narcolepsy UCB Pharma Ltd. 21/02/2005 ORFADIN Nitisinone Treatment of tyrosinaemia type I 21/02/2005 PRIALT Ziconotide (intraspinal use) 16/11/2004 XAGRID Anagrelide Hydrochloride INN=anagrelide 13/10/2004 WILZIN Zinc acetate dihydrate Treatment of chronic pain requiring intraspinal analgesia Treatment of essential thrombocythaemia Treatment of Wilson s disease 29/07/2004 PEDEA Ibuprofen Treatment of a haemodynamically significant patent ductus arteriosus in preterm newborn infants less than 34 weeks of gestational age 28/04/2004 LYSODREN Mitotane Treatment of adrenal cortical 14/04/2004 LITAK Cladribine (subcutaneous use) 25/03/2004 PHOTOBARR Porfimer sodium (for use with photodynamic therapy) Treatment of indolent non- Hodgkin s lymphoma Treatment of high-grade dysplasia in Barrett s Esophagus 17/10/2006 ONSENAL Celecoxib Treatment of Familial Adenomatous Polyposis 16/09/2003 VENTAVIS Iloprost Treatment of primary and of the following forms of secondary pulmonary : connective tissue disease pulmonary, drug-induced pulmonary, portopulmonary, pulmonary associated with congenital heart disease, chronic Swedish Orphan International AB Elan Pharma International Ltd. Shire Pharmaceutical Development Ltd Orphan Europe Orphan Europe Laboratoire HRA Pharma Lipomed GmbH Axcan Pharma International BV Pharmacia-Pfizer EEIG Schering AG
Date (dd/mm/yyyy) 09/07/2003 BUSILVEX Busulfan (Intravenous use) Conditioning treatment prior to hematopoietic progenitor cell transplantation 10/06/2003 ALDURAZYME Laronidase Treatment of Mucopolysaccharidosis, type I 24/01/2003 CARBAGLU N-carbamyl-Lglutamic acid INN=carglumic acid Treatment of N-acetylglutamate synthetase (NAGS) deficiency holder Pierre Fabre Médicament Genzyme B.V. Orphan Europe 20/11/2002 ZAVESCA Miglustat Treatment of Gaucher Disease Actelion Registration 13/11/2002 SOMAVERT Pegvisomant Treatment of acromegaly Pfizer 15/05/2002 TRACLEER Bosentan monohydrate INN=bosentan and chronic Treatment of Systemic Sclerosis 05/03/2002 TRISENOX Arsenic Trioxide Treatment of acute promyelocytic 07/11/2001 GLIVEC Imatinib mesilate Treatment of myelodysplastic / myeloproliferative diseases Treatment of chronic eosinophilic and the hypereosinophilic Treatment of dermatofibrosarcoma protuberans Treatment of chronic myeloid Treatment of malignant gastrointestinal 03/08/2001 FABRAZYME Alpha-Galactosidase A Treatment of Fabry disease INN=agalsidase beta 03/08/2001 REPLAGAL Alpha-Galactosidase A Treatment of Fabry disease INN=agalsidase alfa Actelion Registration Cephalon Uk Ltd Genzyme Europe B.V. Shire Human Genetic Therapies AB
Orphan drugs by therapeutic area Cardiovascular PEDEA Ibuprofen Treatment of a haemodynamically significant patent ductus arteriosus in preterm newborn infants less than 34 weeks of gestational age REVATIO THELIN TRACLEER Sildenafil citrate INN=sildenafil Sitaxentan sodium Bosentan monohydrate INN=bosentan and chronic and chronic and chronic Treatment of Systemic Sclerosis VENTAVIS Iloprost Treatment of primary and of the following forms of secondary pulmonary : connective tissue disease pulmonary, drug-induced pulmonary, portopulmonary, pulmonary associated with congenital heart disease, chronic thromboembolic pulmonary Orphan Europe 29/07/2004 Pfizer 28/10/2005 Encysive (UK) Actelion Registration 10/08/2006 15/05/2002 Schering AG 16/09/2003 Endocrinology INCRELEX Mecasermin Treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects Tercica Europe 03/08/2007 SOMAVERT Pegvisomant Treatment of acromegaly Pfizer 13/11/2002
Gastrointestinal ONSENAL Celecoxib Treatment of Familial Adenomatous Polyposis PHOTOBARR Porfimer sodium (for use with photodynamic therapy) Treatment of high-grade dysplasia in Barrett s Esophagus Pharmacia-Pfizer EEIG Axcan Pharma International BV 17/10/2006 25/03/2004 Hematology SIKLOS Hydroxycarbamide SOLIRIS Eculizumab Treatment of paroxysmal nocturnal haemoglobinuria XAGRID Anagrelide Hydrochloride INN=anagrelide Treatment of sickle cell OTL Pharma 29/06/2007 Treatment of essential thrombocythaemia Alexion Europe SAS Shire Pharmaceutical Development Ltd 20/06/2007 16/11/2004 Immunology BUSILVEX Busulfan (Intravenous use) Conditioning treatment prior to hematopoietic progenitor cell transplantation Pierre Fabre Médicament 09/07/2003
Metabolism ALDURAZYME Laronidase Treatment of Mucopolysaccharidosis, type I CARBAGLU CYSTADANE N-carbamyl-Lglutamic acid INN=carglumic acid Betaine anhydrous Treatment of N-acetylglutamate synthetase (NAGS) deficiency Genzyme B.V. 10/06/2003 Orphan Europe 24/01/2003 Treatment of homocystinuria Orphan Europe 15/02/2007 ELAPRASE Idursulfase Treatment of Mucopolysaccharidosis, type II (Hunter Syndrome) FABRAZYME Alpha-Galactosidase Treatment of Fabry disease A INN=agalsidase beta MYOZYME NAGLAZYME Recombinant human acid alpha-glucosidase INN=alglucosidase alfa N-acetylgalactosamine-4-sulfatase INN=galsulfase Treatment of Glycogen Storage Disease type II (Pompe s disease) Treatment of Mucopolysaccharidosis, type VI (Maroteaux-Lamy Syndrome) TKT UK Ltd 08/01/2007 Genzyme Europe B.V. Genzyme Europe B.V. BioMarin Europe Ltd 03/08/2001 29/03/2006 24/01/2006 ORFADIN Nitisinone Treatment of tyrosinaemia type I Swedish Orphan 21/02/2005 International AB REPLAGAL Alpha-Galactosidase Treatment of Fabry disease TKT UK Ltd. 03/08/2001 A INN=agalsidase alfa WILZIN Zinc acetate dihydrate Treatment of Wilson s disease Orphan Europe 13/10/2004 ZAVESCA Miglustat Treatment of Gaucher Disease Actelion Registration 20/11/2002 Neurology DIACOMIT Stiripentol Treatment of severe myoclonic epilepsy in infancy INOVELON Rufinamide Treatment of Lennox-Gastaut PRIALT XYREM Ziconotide (intraspinal use) Sodium oxybate Treatment of chronic pain requiring intraspinal analgesia Biocodex 04/01/2007 Eisai 16/01/2007 Elan Pharma International Ltd. 21/02/2005 Treatment of narcolepsy UCB Pharma Ltd. 13/10/2004 10
Oncology ATRIANCE Nelarabine EVOLTRA Clofarabine GLIOLAN GLIVEC LITAK 5-aminolevulinic acid hydrochloride Imatinib mesilate Cladribine (subcutaneous use) Intra-operative photodynamic diagnosis of residual glioma Treatment of chronic myeloid Treatment of myelodysplastic / myeloproliferative diseases Treatment of chronic eosinophilic and the hypereosinophilic Treatment of dermatofibrosarcoma protuberans Treatment of malignant gastrointestinal Treatment of indolent non- Hodgkin s lymphoma LYSODREN Mitotane Treatment of adrenal cortical GlaxoSmithKline Research & Development Bioenvision Medac Gesellschaft für klinische Spezialpräparate mbh 22/08/2007 29/05/2006 07/09/2007 07/11/2001 Lipomed GmbH 14/04/2004 Laboratoire HRA Pharma NEXAVAR Sorafenib Treatment of renal cell Bayer Healthcare AG Treatment of hepatocellular REVLIMID Lenalidomide Treatment of Multiple Myeloma Celgene Europe SPRYCEL Dasatinib Treatment of chronic myeloid SUTENT Sunitinib malate INN=sunitinib Treatment of malignant gastrointestinal Treatment of renal cell TASIGNA Nilotinib Treatment of chronic myeloid Bristol-Myers Squibb Pharma EEIG 28/04/2004 19/07/2006 14/06/2007 20/11/2006 Pfizer 19/07/2006 TORISEL Temsirolimus Treatment of renal cell Wyeth Europa TRISENOX Arsenic Trioxide Treatment of acute promyelocytic Cell Therapeutics (UK) 19/11/2007 19/11/2007 05/03/2002 YONDELIS Trabectedin Treatment of soft tissue sarcoma Pharma Mar SA 17/09/2007 11
Toxicology Tradename Active substance Designated orphan EXJADE Deferasirox Chronic iron overload requiring chelation therapy SAVENE Dexrazoxane Treatment of anthracycline extravasations 28/08/2006 TopoTarget A/S 28/07/2006 12
authorisation holder holder Actelion Registration TRACLEER Bosentan monohydrate INN=bosentan Actelion Registration Addmedica SAS SIKLOS Hydroxycarbamide and chronic Treatment of Systemic Sclerosis 15/05/2002 ZAVESCA Miglustat Treatment of Gaucher Disease 20/11/2002 Treatment of sickle cell Alexion Europe SAS SOLIRIS Eculizumab Treatment of paroxysmal nocturnal haemoglobinuria Axcan Pharma International BV PHOTOBARR Porfimer sodium (for use with photodynamic therapy) Treatment of high-grade dysplasia in Barrett s Esophagus Bayer Healthcare AG NEXAVAR Sorafenib Treatment of renal cell Treatment of hepatocellular Biocodex DIACOMIT Stiripentol Treatment of severe myoclonic epilepsy in infancy Bioenvision EVOLTRA Clofarabine BioMarin Europe Ltd NAGLAZYME N-acetylgalactosamine-4-sulfatase INN=galsulfase Bristol-Myers Squibb Pharma EEIG Celgene Europe Treatment of Mucopolysaccharidosis, type VI (Maroteaux-Lamy Syndrome) SPRYCEL Dasatinib Treatment of chronic myeloid REVLIMID Lenalidomide Treatment of Multiple Myeloma Cephalon Uk Ltd TRISENOX Arsenic Trioxide Treatment of acute promyelocytic Eisai INOVELON Rufinamide Treatment of Lennox-Gastaut Elan Pharma International Ltd. PRIALT Ziconotide (intraspinal use) Encysive (UK) THELIN Sitaxentan sodium Treatment of chronic pain requiring intraspinal analgesia and chronic 29/06/2007 20/06/2007 25/03/2004 19/07/2006 04/01/2007 29/05/2006 24/01/2006 20/11/2006 14/06/2007 05/03/2002 16/01/2007 21/02/2005 10/08/2006 Genzyme B.V. ALDURAZYME Laronidase Treatment of Mucopolysaccharidosis, 10/06/2003 type I Genzyme Europe B.V. FABRAZYME Alpha-Galactosidase A INN=agalsidase beta Treatment of Fabry disease 03/08/2001 13
holder Genzyme Europe B.V. MYOZYME Recombinant human acid alpha-glucosidase INN=alglucosidase alfa Treatment of Glycogen Storage Disease type II (Pompe s disease) Glaxo Group ATRIANCE Nelarabine Laboratoire HRA Pharma LYSODREN Mitotane Treatment of adrenal cortical Lipomed GmbH LITAK Cladribine (subcutaneous use) Medac GmbH GLIOLAN 5-aminolevulinic acid hydrochloride Treatment of indolent non- Hodgkin s lymphoma Intra-operative photodynamic diagnosis of residual glioma EXJADE Deferasirox Chronic iron overload requiring chelation therapy GLIVEC Imatinib mesilate Treatment of malignant gastrointestinal Treatment of myelodysplastic / myeloproliferative diseases Treatment of chronic eosinophilic and the hypereosinophilic Treatment of dermatofibrosarcoma protuberans Treatment of chronic myeloid TASIGNA Nilotinib Treatment of chronic myeloid Orphan Europe CARBAGLU N-carbamyl-Lglutamic acid INN=carglumic acid Orphan Europe CYSTADANE Betaine anhydrous 29/03/2006 22/08/2007 28/04/2004 14/04/2004 07/09/2007 28/08/2006 07/11/2001 19/11/2007 Treatment of N-acetylglutamate synthetase (NAGS) 24/01/2003 deficiency Treatment of homocystinuria 15/02/2007 Orphan Europe PEDEA Ibuprofen Treatment of a haemodynamically significant patent ductus arteriosus in preterm newborn infants less than 34 weeks of gestational age Orphan Europe WILZIN Zinc acetate dihydrate Pfizer REVATIO Sildenafil citrate INN=sildenafil Treatment of Wilson s disease and chronic 29/07/2004 13/10/2004 28/10/2005 Pfizer SOMAVERT Pegvisomant Treatment of acromegaly 13/11/2002 Pfizer SUTENT Sunitinib malate Treatment of malignant gastrointestinal 19/07/2006 INN=sunitinib Treatment of renal cell 14
holder Pharmacia-Pfizer EEIG ONSENAL Celecoxib Treatment of Familial Adenomatous Polyposis Pharma Mar SA YONDELIS Trabectedin Treatment of soft tissue sarcoma Pierre Fabre Médicament BUSILVEX Busulfan (Intravenous use) Conditioning treatment prior to hematopoietic progenitor cell transplantation Schering AG VENTAVIS Iloprost Treatment of primary and of the following forms of secondary pulmonary : connective tissue disease pulmonary, drug-induced pulmonary, portopulmonary, pulmonary associated with congenital heart disease, chronic Shire Human Genetic Therapies AB Shire Human Genetic Therapies AB Shire Pharmaceutical Development Ltd Swedish Orphan International AB Tercica Europe 17/10/2006 17/09/2007 09/07/2003 16/09/2003 ELAPRASE Idursulfase Treatment of Mucopolysaccharidosis, type II (Hunter Syndrome) 08/01/2007 REPLAGAL Alpha-Galactosidase A Treatment of Fabry disease 03/08/2001 INN=agalsidase alfa XAGRID Anagrelide Hydrochloride INN=anagrelide Treatment of essential thrombocythaemia ORFADIN Nitisinone Treatment of tyrosinaemia type I INCRELEX Mecasermin Treatment of primary insulinlike growth factor-1 deficiency due to molecular or genetic defects 16/11/2004 21/02/2005 03/08/2007 TopoTarget A/S SAVENE Dexrazoxane Treatment of anthracycline 28/07/2006 extravasations UCB Pharma Ltd. XYREM Sodium oxybate Treatment of narcolepsy 13/10/2004 Wyeth Europa TORISEL Temsirolimus Treatment of renal cell 19/11/2007 15