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Human medicine European public assessment report (EPAR): Qinlock
ripretinib, Gastrointestinal Stromal Tumors
Date of authorisation: 18/11/2021,
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, Revision: 5, Authorised, Last updated: 30/08/2023
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Human medicine European public assessment report (EPAR): Zolgensma
onasemnogene abeparvovec, Muscular Atrophy, Spinal
Date of authorisation: 18/05/2020,, , 
, Revision: 12, Authorised, Last updated: 29/08/2023
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Human medicine European public assessment report (EPAR): Gavreto
pralsetinib, Carcinoma, Non-Small-Cell Lung
Date of authorisation: 18/11/2021,, , Revision: 6, Authorised, Last updated: 28/08/2023
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Human medicine European public assessment report (EPAR): Enjaymo
sutimlimab, Hemolysis; Anemia, Hemolytic, Autoimmune
Date of authorisation: 15/11/2022,, , Revision: 3, Authorised, Last updated: 28/08/2023
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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: 5, Authorised, Last updated: 28/08/2023
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Human medicine European public assessment report (EPAR): Cerdelga
eliglustat, Gaucher Disease
Date of authorisation: 19/01/2015,, , Revision: 16, Authorised, Last updated: 28/08/2023
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Human medicine European public assessment report (EPAR): Kanuma
sebelipase alfa, Lipid Metabolism, Inborn Errors
Date of authorisation: 28/08/2015,, 
, , Revision: 10, Authorised, Last updated: 25/08/2023
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Human medicine European public assessment report (EPAR): Artesunate Amivas
artesunate, Malaria
Date of authorisation: 22/11/2021,, , Revision: 2, Authorised, Last updated: 23/08/2023
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Human medicine European public assessment report (EPAR): Kinpeygo
budesonide, micronised, Glomerulonephritis, IGA
Date of authorisation: 15/07/2022,, , , Revision: 5, Authorised, Last updated: 21/08/2023
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Human medicine European public assessment report (EPAR): Soliris
Eculizumab, Hemoglobinuria, Paroxysmal
Date of authorisation: 20/06/2007,, , Revision: 36, Authorised, Last updated: 21/08/2023
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Human medicine European public assessment report (EPAR): Caprelsa
Vandetanib, Thyroid Neoplasms
Date of authorisation: 16/02/2012,, Revision: 52, Authorised, Last updated: 16/08/2023
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Human medicine European public assessment report (EPAR): Takhzyro
lanadelumab, Angioedemas, Hereditary
Date of authorisation: 22/11/2018,, , Revision: 11, Authorised, Last updated: 16/08/2023
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Human medicine European public assessment report (EPAR): Deltyba
Delamanid, Tuberculosis, Multidrug-Resistant
Date of authorisation: 27/04/2014,
Date of refusal: 25/07/2013,, , , Revision: 25, Authorised, Last updated: 14/08/2023
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Human medicine European public assessment report (EPAR): Arikayce liposomal
Amikacin sulfate, Respiratory Tract Infections
Date of authorisation: 27/10/2020,, Revision: 2, Authorised, Last updated: 04/08/2023
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Human medicine European public assessment report (EPAR): Idefirix
Imlifidase, Desensitization, Immunologic; Kidney Transplantation
Date of authorisation: 25/08/2020,, , , Revision: 4, Authorised, Last updated: 01/08/2023
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Human medicine European public assessment report (EPAR): Sogroya
Somapacitan, Growth
Date of authorisation: 31/03/2021,, , Revision: 2, Authorised, Last updated: 31/07/2023
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Human medicine European public assessment report (EPAR): Ztalmy
Ganaxolone, Epileptic Syndromes; Spasms, Infantile
Date of authorisation: 26/07/2023,, , Authorised, Last updated: 31/07/2023
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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: 7, Authorised, Last updated: 28/07/2023
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Human medicine European public assessment report (EPAR): Abecma
idecabtagene vicleucel, Multiple Myeloma; Neoplasms; Cancer; Neoplasms, Plasma Cell; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Infectious Mononucleosis; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases
Date of authorisation: 18/08/2021,, , , Revision: 6, Authorised, Last updated: 27/07/2023
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Human medicine European public assessment report (EPAR): Tecvayli
Teclistamab, Multiple Myeloma
Date of authorisation: 23/08/2022,, , Revision: 1, Authorised, Last updated: 27/07/2023
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Human medicine European public assessment report (EPAR): Revestive
teduglutide, Malabsorption Syndromes
Date of authorisation: 30/08/2012,, , Revision: 22, Authorised, Last updated: 27/07/2023
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Human medicine European public assessment report (EPAR): Kaftrio
ivacaftor, tezacaftor, elexacaftor, Cystic Fibrosis
Date of authorisation: 21/08/2020,, , Revision: 15, Authorised, Last updated: 26/07/2023
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Human medicine European public assessment report (EPAR): Carvykti
ciltacabtagene autoleucel, Multiple Myeloma
Date of authorisation: 25/05/2022,, , , Revision: 4, Authorised, Last updated: 26/07/2023
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Human medicine European public assessment report (EPAR): Rydapt
Midostaurin, Leukemia, Myeloid, Acute; Mastocytosis
Date of authorisation: 18/09/2017,, Revision: 11, Authorised, Last updated: 26/07/2023
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Human medicine European public assessment report (EPAR): Nulibry
fosdenopterin hydrobromide dihydrate, Metal Metabolism, Inborn Errors
Date of authorisation: 15/09/2022,, , Revision: 2, Authorised, Last updated: 21/07/2023