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163 results
Medicine type
Orphan medicine Remove Orphan medicine filter
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Human medicine European public assessment report (EPAR): Symkevi
tezacaftor, ivacaftor, Cystic Fibrosis
Date of authorisation: 31/10/2018,
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, Revision: 11, Authorised, Last updated: 24/03/2023
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Human medicine European public assessment report (EPAR): Opsumit
Macitentan, Hypertension, Pulmonary
Date of authorisation: 20/12/2013,, Revision: 22, Authorised, Last updated: 24/03/2023
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Human medicine European public assessment report (EPAR): Zokinvy
Lonafarnib, Progeria; Laminopathies
Date of authorisation: 18/07/2022,, , 
, Revision: 1, Authorised, Last updated: 24/03/2023
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Human medicine European public assessment report (EPAR): Enspryng
satralizumab, Neuromyelitis Optica
Date of authorisation: 24/06/2021,, Revision: 2, Authorised, Last updated: 24/03/2023
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Human medicine European public assessment report (EPAR): Polivy
polatuzumab vedotin, Lymphoma, B-Cell
Date of authorisation: 16/01/2020,, , 
, Revision: 5, Authorised, Last updated: 22/03/2023
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Human medicine European public assessment report (EPAR): Darzalex
Daratumumab, Multiple Myeloma
Date of authorisation: 20/05/2016,
, , Revision: 20, Authorised, Last updated: 22/03/2023
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Human medicine European public assessment report (EPAR): Imcivree
Setmelanotide, Obesity
Date of authorisation: 16/07/2021,, , Revision: 7, Authorised, Last updated: 22/03/2023
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Human medicine European public assessment report (EPAR): Inrebic
fedratinib dihydrochloride monohydrate, Myeloproliferative Disorders; Primary Myelofibrosis
Date of authorisation: 08/02/2021,, , Revision: 4, Authorised, Last updated: 21/03/2023
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Human medicine European public assessment report (EPAR): Reblozyl
Luspatercept, Anemia; Myelodysplastic Syndromes; beta-Thalassemia
Date of authorisation: 25/06/2020,, , Revision: 4, Authorised, Last updated: 20/03/2023
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Human medicine European public assessment report (EPAR): Isturisa
Osilodrostat phosphate, Cushing Syndrome
Date of authorisation: 09/01/2020,, , Revision: 4, Authorised, Last updated: 20/03/2023
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Human medicine European public assessment report (EPAR): Livtencity
Maribavir, Cytomegalovirus Infections
Date of authorisation: 09/11/2022,, , Revision: 3, Authorised, Last updated: 17/03/2023
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Human medicine European public assessment report (EPAR): Elzonris
tagraxofusp, Lymphoma
Date of authorisation: 07/01/2021,, , , Revision: 6, Authorised, Last updated: 15/03/2023
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Human medicine European public assessment report (EPAR): Ayvakyt
Avapritinib, Gastrointestinal Stromal Tumors
Date of authorisation: 24/09/2020,, , , Revision: 6, Authorised, Last updated: 14/03/2023
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Human medicine European public assessment report (EPAR): Adcetris
Brentuximab vedotin, Lymphoma, Non-Hodgkin; Hodgkin Disease
Date of authorisation: 25/10/2012,, Revision: 34, Authorised, Last updated: 13/03/2023
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Human medicine European public assessment report (EPAR): Ebvallo
Tabelecleucel, Lymphoproliferative Disorders
Date of authorisation: 16/12/2022,, , , Revision: 2, Authorised, Last updated: 10/03/2023
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Human medicine European public assessment report (EPAR): Prevymis
Letermovir, Cytomegalovirus Infections
Date of authorisation: 08/01/2018,, Revision: 14, Authorised, Last updated: 08/03/2023
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Human medicine European public assessment report (EPAR): SomaKit TOC
edotreotide, Neuroendocrine Tumors; Radionuclide Imaging
Date of authorisation: 08/12/2016,, Revision: 14, Authorised, Last updated: 07/03/2023
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Human medicine European public assessment report (EPAR): Scemblix
asciminib hydrochloride, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Date of authorisation: 25/08/2022,, , Revision: 2, Authorised, Last updated: 02/03/2023
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Human medicine European public assessment report (EPAR): Vyndaqel
tafamidis, Amyloidosis
Date of authorisation: 16/11/2011,, , , Revision: 25, Authorised, Last updated: 02/03/2023
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Human medicine European public assessment report (EPAR): Revestive
teduglutide, Malabsorption Syndromes
Date of authorisation: 30/08/2012,, , Revision: 21, Authorised, Last updated: 01/03/2023
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Human medicine European public assessment report (EPAR): Dovprela (previously Pretomanid FGK)
Pretomanid, Tuberculosis, Multidrug-Resistant
Date of authorisation: 31/07/2020,, , , Revision: 7, Authorised, Last updated: 24/02/2023
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Human medicine European public assessment report (EPAR): Lamzede
velmanase alfa, alpha-Mannosidosis
Date of authorisation: 23/03/2018,, , , Revision: 6, Authorised, Last updated: 22/02/2023
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Human medicine European public assessment report (EPAR): Natpar
parathyroid hormone, Hypoparathyroidism
Date of authorisation: 24/04/2017,, , , Revision: 14, Authorised, Last updated: 22/02/2023
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Human medicine European public assessment report (EPAR): Namuscla
Mexiletine hydrochloride, Myotonic Disorders
Date of authorisation: 18/12/2018,, Revision: 5, Authorised, Last updated: 22/02/2023
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Human medicine European public assessment report (EPAR): Oxbryta
Voxelotor, Anemia; Anemia, Hemolytic; Anemia, Sickle Cell
Date of authorisation: 14/02/2022,, , Revision: 2, Authorised, Last updated: 20/02/2023