For help on how to get the results you want, see our search tips.
463 results
Medicine type
Orphan medicine Remove Orphan medicine filter
-
List item
Human medicine European public assessment report (EPAR): Kanuma (updated)
sebelipase alfa, Lipid Metabolism, Inborn Errors
Date of authorisation: 28/08/2015,
, 
, 
, Revision: 5, Authorised, Last updated: 01/12/2020
-
List item
Human medicine European public assessment report (EPAR): Mylotarg (updated)
gemtuzumab ozogamicin, Leukemia, Myeloid, Acute
Date of authorisation: 19/04/2018,, , Revision: 6, Authorised, Last updated: 30/11/2020
-
List item
Human medicine European public assessment report (EPAR): Scenesse (updated)
afamelanotide, Protoporphyria, Erythropoietic
Date of authorisation: 22/12/2014,, , 
, Revision: 7, Authorised, Last updated: 30/11/2020
-
List item
Human medicine European public assessment report (EPAR): Aplidin (updated)
, Last updated: 26/11/2020
-
List item
Human medicine European public assessment report (EPAR): Lamzede (updated)
velmanase alfa, alpha-Mannosidosis
Date of authorisation: 23/03/2018,, , , Revision: 3, Authorised, Last updated: 24/11/2020
-
List item
Human medicine European public assessment report (EPAR): Alofisel (updated)
darvadstrocel, Rectal Fistula
Date of authorisation: 23/03/2018,, , Revision: 5, Authorised, Last updated: 24/11/2020
-
List item
Human medicine European public assessment report (EPAR): Vyndaqel (updated)
tafamidis, Amyloidosis
Date of authorisation: 16/11/2011,, , , Revision: 17, Authorised, Last updated: 23/11/2020
-
List item
Human medicine European public assessment report (EPAR): Darzalex (updated)
Daratumumab, Multiple Myeloma
Date of authorisation: 20/05/2016,, , , Revision: 14, Authorised, Last updated: 20/11/2020
-
List item
Human medicine European public assessment report (EPAR): Cablivi (updated)
Caplacizumab, Purpura, Thrombotic Thrombocytopenic
Date of authorisation: 30/08/2018,, , Revision: 4, Authorised, Last updated: 20/11/2020
-
List item
Human medicine European public assessment report (EPAR): Myalepta (updated)
Metreleptin, Lipodystrophy, Familial Partial
Date of authorisation: 29/07/2018,, , , Revision: 4, Authorised, Last updated: 20/11/2020
-
List item
Human medicine European public assessment report (EPAR): Polivy (updated)
polatuzumab vedotin, Lymphoma, B-Cell
Date of authorisation: 16/01/2020,, , 
, Revision: 1, Authorised, Last updated: 19/11/2020
-
List item
Human medicine European public assessment report (EPAR): Procysbi (updated)
mercaptamine bitartrate, Cystinosis
Date of authorisation: 05/09/2013,, Revision: 13, Authorised, Last updated: 17/11/2020
-
List item
Human medicine European public assessment report (EPAR): Besponsa (updated)
inotuzumab ozogamicin, Precursor Cell Lymphoblastic Leukemia-Lymphoma
Date of authorisation: 28/06/2017,, , Revision: 7, Authorised, Last updated: 16/11/2020
-
List item
Human medicine European public assessment report (EPAR): Xermelo (updated)
telotristat etiprate, Carcinoid Tumor, Neuroendocrine Tumors
Date of authorisation: 17/09/2017,, , Revision: 11, Authorised, Last updated: 11/11/2020
-
List item
Human medicine European public assessment report (EPAR): Zolgensma (updated)
onasemnogene abeparvovec, Muscular Atrophy, Spinal
Date of authorisation: 18/05/2020,, , , Revision: 1, Authorised, Last updated: 09/11/2020
-
List item
Human medicine European public assessment report (EPAR): Ayvakyt (updated)
avapritinib, Gastrointestinal Stromal Tumors
Date of authorisation: 24/09/2020,, , Revision: 1, Authorised, Last updated: 06/11/2020
-
List item
Human medicine European public assessment report (EPAR): Zejula (updated)
Niraparib (tosylate monohydrate), Fallopian Tube Neoplasms, Peritoneal Neoplasms, Ovarian Neoplasms
Date of authorisation: 16/11/2017,, , Revision: 11, Authorised, Last updated: 06/11/2020
-
List item
Human medicine European public assessment report (EPAR): Defitelio (updated)
defibrotide, Hepatic Veno-Occlusive Disease
Date of authorisation: 18/10/2013,, , , Revision: 12, Authorised, Last updated: 05/11/2020
-
List item
Human medicine European public assessment report (EPAR): Crysvita (updated)
Burosumab, Hypophosphatemia, Familial, Hypophosphatemic Rickets, X-Linked Dominant
Date of authorisation: 19/02/2018,, , , Revision: 6, Authorised, Last updated: 04/11/2020
-
List item
Human medicine European public assessment report (EPAR): Alprolix (updated)
eftrenonacog alfa, Hemophilia B
Date of authorisation: 12/05/2016,, , Revision: 5, Authorised, Last updated: 04/11/2020
-
List item
Human medicine European public assessment report (EPAR): NexoBrid
concentrate of proteolytic enzymes enriched in bromelain, Debridement
Date of authorisation: 18/12/2012,, , Revision: 10, Authorised, Last updated: 03/11/2020
-
List item
Human medicine European public assessment report (EPAR): Spinraza
nusinersen sodium, Muscular Atrophy, Spinal
Date of authorisation: 30/05/2017,, , , Revision: 10, Authorised, Last updated: 03/11/2020
-
List item
Human medicine European public assessment report (EPAR): Palynziq
Pegvaliase, Phenylketonurias
Date of authorisation: 03/05/2019,, , Revision: 1, Authorised, Last updated: 30/10/2020
-
List item
Human medicine European public assessment report (EPAR): Hetlioz
tasimelteon, Sleep Disorders, Circadian Rhythm
Date of authorisation: 03/07/2015,, Revision: 6, Authorised, Last updated: 29/10/2020
-
List item
Human medicine European public assessment report (EPAR): Bronchitol
mannitol, Cystic Fibrosis
Date of authorisation: 13/04/2012,, Revision: 15, Authorised, Last updated: 28/10/2020