531 results
Medicine type
Orphan medicine Remove Orphan medicine filter
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Human medicine European public assessment report (EPAR): Defitelio (updated)
defibrotide, Hepatic Veno-Occlusive Disease
Date of authorisation: 18/10/2013,
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, Revision: 7, Authorised
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Human medicine European public assessment report (EPAR): Myalepta (updated)
Metreleptin, Lipodystrophy, Familial Partial
Date of authorisation: 29/07/2018,, , , Revision: 2, Authorised
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Human medicine European public assessment report (EPAR): Verkazia (updated)
ciclosporin, Conjunctivitis, Keratitis
Date of authorisation: 06/07/2018,, Revision: 1, Authorised
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Human medicine European public assessment report (EPAR): Kalydeco (updated)
ivacaftor, Cystic Fibrosis
Date of authorisation: 22/07/2012,, Revision: 17, Authorised
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Human medicine European public assessment report (EPAR): Xaluprine (previously Mercaptopurine Nova Laboratories) (updated)
6-mercaptopurine monohydrate, Leukemia, Lymphoid
Date of authorisation: 09/03/2012,, Revision: 8, Authorised
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Human medicine European public assessment report (EPAR): Somavert (updated)
Pegvisomant, Acromegaly
Date of authorisation: 12/11/2002,, Revision: 20, Authorised
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Human medicine European public assessment report (EPAR): Luxturna (updated)
voretigene neparvovec, Leber Congenital Amaurosis, Retinitis Pigmentosa
Date of authorisation: 22/11/2018,, , Revision: 1, Authorised
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Human medicine European public assessment report (EPAR): Esbriet (updated)
Pirfenidone, Idiopathic Pulmonary Fibrosis
Date of authorisation: 27/02/2011,, Revision: 23, Authorised
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Human medicine European public assessment report (EPAR): Holoclar (updated)
ex vivo expanded autologous human corneal epithelial cells containing stem cells, Stem Cell Transplantation, Corneal Diseases
Date of authorisation: 17/02/2015,, , 
, Revision: 5, Authorised
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Human medicine European public assessment report (EPAR): Siklos (updated)
hydroxycarbamide, Anemia, Sickle Cell
Date of authorisation: 29/06/2007,, Revision: 16, Authorised
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Human medicine European public assessment report (EPAR): Mepact (updated)
mifamurtide, Osteosarcoma
Date of authorisation: 06/03/2009,, Revision: 14, Authorised
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Human medicine European public assessment report (EPAR): Alprolix (updated)
eftrenonacog alfa, Hemophilia B
Date of authorisation: 12/05/2016,, , Revision: 4, Authorised
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Human medicine European public assessment report (EPAR): Cometriq (updated)
cabozantinib, Thyroid Neoplasms
Date of authorisation: 21/03/2014,, , , Revision: 13, Authorised
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Human medicine European public assessment report (EPAR): Coagadex (updated)
Human coagulation factor X, Factor X Deficiency
Date of authorisation: 16/03/2016,, , Revision: 3, Authorised
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Human medicine European public assessment report (EPAR): Deltyba (updated)
Delamanid, Tuberculosis, Multidrug-Resistant
Date of authorisation: 27/04/2014,
Date of refusal: 25/07/2013,, , , Revision: 11, Authorised
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Human medicine European public assessment report (EPAR): Bronchitol (updated)
mannitol, Cystic Fibrosis
Date of authorisation: 13/04/2012,, Revision: 13, Authorised
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Human medicine European public assessment report (EPAR): Spinraza (updated)
nusinersen sodium, Muscular Atrophy, Spinal
Date of authorisation: 30/05/2017,, , Revision: 6, Authorised
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Human medicine European public assessment report (EPAR): Lutathera (updated)
lutetium (177Lu) oxodotreotide, Neuroendocrine Tumors
Date of authorisation: 26/09/2017,, , Revision: 2, Authorised
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Human medicine European public assessment report (EPAR): Galafold (updated)
migalastat hydrochloride, Fabry Disease
Date of authorisation: 25/05/2016,, , Revision: 8, Authorised
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Human medicine European public assessment report (EPAR): Cresemba (updated)
isavuconazole, Aspergillosis
Date of authorisation: 15/10/2015,, , Revision: 5, Authorised
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Human medicine European public assessment report (EPAR): Carbaglu (updated)
carglumic acid, Amino Acid Metabolism, Inborn Errors, Propionic Acidemia
Date of authorisation: 24/01/2003,, Revision: 17, Authorised
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Human medicine European public assessment report (EPAR): Symkevi
tezacaftor, ivacaftor, Cystic Fibrosis
Date of authorisation: 31/10/2018,, , Revision: 1, Authorised
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Human medicine European public assessment report (EPAR): Scenesse
afamelanotide, Protoporphyria, Erythropoietic
Date of authorisation: 22/12/2014,, , , Revision: 3, Authorised
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Human medicine European public assessment report (EPAR): Mepsevii
vestronidase alfa, Mucopolysaccharidosis VII
Date of authorisation: 23/08/2018,, , , Revision: 1, Authorised
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Human medicine European public assessment report (EPAR): Qarziba (previously Dinutuximab beta EUSA and Dinutuximab beta Apeiron)
dinutuximab beta, Neuroblastoma
Date of authorisation: 08/05/2017,, , , Revision: 5, Authorised