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Human medicine European public assessment report (EPAR): Ayvakyt
avapritinib, Gastrointestinal Stromal Tumors
Date of authorisation: 24/09/2020,
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, Authorised, Last updated: 30/09/2020
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Human medicine European public assessment report (EPAR): Blenrep
belantamab mafodotin, Multiple Myeloma
Date of authorisation: 25/08/2020,
, , , Authorised, Last updated: 17/09/2020
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Human medicine European public assessment report (EPAR): Idefirix
Imlifidase, Desensitization, Immunologic, Kidney Transplantation
Date of authorisation: 25/08/2020,, , , Authorised, Last updated: 28/08/2020
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Human medicine European public assessment report (EPAR): Kaftrio (updated)
ivacaftor / tezacaftor / elexacaftor, Cystic Fibrosis
Date of authorisation: 21/08/2020,, , Authorised, Last updated: 29/09/2020
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Human medicine European public assessment report (EPAR): Pretomanid FGK
Pretomanid, Tuberculosis, Multidrug-Resistant
Date of authorisation: 31/07/2020,, , , Authorised, Last updated: 11/08/2020
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Human medicine European public assessment report (EPAR): Hepcludex (updated)
Bulevirtide acetate, Hepatitis D, Chronic
Date of authorisation: 31/07/2020,, Revision: 1, Authorised, Last updated: 07/10/2020
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Human medicine European public assessment report (EPAR): Daurismo
Glasdegib maleate, Leukemia, Myeloid, Acute
Date of authorisation: 26/06/2020,, , Authorised, Last updated: 10/07/2020
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Human medicine European public assessment report (EPAR): Reblozyl
Luspatercept, Anemia, Myelodysplastic Syndromes, beta-Thalassemia
Date of authorisation: 25/06/2020,, , Authorised, Last updated: 08/07/2020
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Human medicine European public assessment report (EPAR): Zolgensma
onasemnogene abeparvovec, Muscular Atrophy, Spinal
Date of authorisation: 18/05/2020,, , , Authorised, Last updated: 27/05/2020
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Human medicine European public assessment report (EPAR): Trepulmix
Treprostinil sodium, Hypertension, Pulmonary
Date of authorisation: 03/04/2020,, Authorised, Last updated: 08/04/2020
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Human medicine European public assessment report (EPAR): Givlaari
Givosiran, Porphyrias, Hepatic
Date of authorisation: 02/03/2020,, , Authorised, Last updated: 09/03/2020
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Human medicine European public assessment report (EPAR): Polivy
polatuzumab vedotin, Lymphoma, B-Cell
Date of authorisation: 16/01/2020,, , , Authorised, Last updated: 27/01/2020
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Human medicine European public assessment report (EPAR): Isturisa
Osilodrostat phosphate, Cushing Syndrome
Date of authorisation: 09/01/2020,, , Revision: 2, Authorised, Last updated: 16/06/2020
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Human medicine European public assessment report (EPAR): Xospata
gilteritinib fumarate, Leukemia, Myeloid, Acute
Date of authorisation: 24/10/2019,, , Authorised, Last updated: 08/11/2019
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Human medicine European public assessment report (EPAR): Epidyolex
Cannabidiol, Lennox Gastaut Syndrome, Epilepsies, Myoclonic
Date of authorisation: 19/09/2019,, Revision: 4, Authorised, Last updated: 18/09/2020
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Human medicine European public assessment report (EPAR): Zynteglo
Autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiglobin BB305 lentiviral vector encoding the beta-A-T87Q-globin gene, beta-Thalassemia
Date of authorisation: 29/05/2019,, 
, , , Revision: 2, Authorised, Last updated: 06/05/2020
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Human medicine European public assessment report (EPAR): Waylivra
Volanesorsen sodium, Hyperlipoproteinemia Type I
Date of authorisation: 03/05/2019,, , , Revision: 2, Authorised, Last updated: 19/05/2020
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Human medicine European public assessment report (EPAR): Palynziq
Pegvaliase, Phenylketonurias
Date of authorisation: 03/05/2019,, , Authorised, Last updated: 29/05/2019
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Human medicine European public assessment report (EPAR): Namuscla
mexiletine hcl, Myotonic Disorders
Date of authorisation: 18/12/2018,, Revision: 3, Authorised, Last updated: 27/07/2020
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Human medicine European public assessment report (EPAR): Poteligeo
Mogamulizumab, Sezary Syndrome, Mycosis Fungoides
Date of authorisation: 22/11/2018,, , Revision: 1, Authorised, Last updated: 10/01/2020
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Human medicine European public assessment report (EPAR): Luxturna
voretigene neparvovec, Leber Congenital Amaurosis, Retinitis Pigmentosa
Date of authorisation: 22/11/2018,, , Revision: 2, Authorised, Last updated: 30/01/2020
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Human medicine European public assessment report (EPAR): Takhzyro
lanadelumab, Angioedemas, Hereditary
Date of authorisation: 22/11/2018,, , , Revision: 3, Authorised, Last updated: 03/09/2020
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Human medicine European public assessment report (EPAR): Symkevi
tezacaftor, ivacaftor, Cystic Fibrosis
Date of authorisation: 31/10/2018,, , Revision: 6, Authorised, Last updated: 26/06/2020
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Human medicine European public assessment report (EPAR): Cablivi
Caplacizumab, Purpura, Thrombotic Thrombocytopenic
Date of authorisation: 30/08/2018,, , Revision: 3, Authorised, Last updated: 22/06/2020
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Human medicine European public assessment report (EPAR): Onpattro
patisiran sodium, Amyloidosis, Familial
Date of authorisation: 27/08/2018,, , , Revision: 6, Authorised, Last updated: 08/09/2020