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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): Pemazyre (updated)
pemigatinib, Cholangiocarcinoma
Date of authorisation: 26/03/2021,
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, Revision: 5, Authorised, Last updated: 02/03/2023
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Human medicine European public assessment report (EPAR): Scemblix (updated)
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): Onpattro
patisiran sodium, Amyloidosis, Familial
Date of authorisation: 27/08/2018,, 
, , Revision: 10, Authorised, Last updated: 28/02/2023
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Human medicine European public assessment report (EPAR): Hemgenix
etranacogene dezaparvovec, Hemophilia B
Date of authorisation: 20/02/2023,, , , Authorised, Last updated: 28/02/2023
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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): Qinlock
ripretinib, Gastrointestinal Stromal Tumors
Date of authorisation: 18/11/2021,, , Revision: 2, Authorised, Last updated: 23/02/2023
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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
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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: 13, Authorised, Last updated: 20/02/2023
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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: 1, Authorised, Last updated: 20/02/2023
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Human medicine European public assessment report (EPAR): Amglidia
glibenclamide, Diabetes Mellitus
Date of authorisation: 24/05/2018,, Revision: 5, Authorised, Last updated: 16/02/2023
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Human medicine European public assessment report (EPAR): Strensiq
asfotase alfa, Hypophosphatasia
Date of authorisation: 28/08/2015,, , , Revision: 16, Authorised, Last updated: 15/02/2023
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Human medicine European public assessment report (EPAR): NexoBrid
proteolytic enzymes enriched in bromelain, Debridement
Date of authorisation: 18/12/2012,, Revision: 14, Authorised, Last updated: 15/02/2023
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Human medicine European public assessment report (EPAR): Zejula
Niraparib (tosilate monohydrate), Fallopian Tube Neoplasms; Peritoneal Neoplasms; Ovarian Neoplasms
Date of authorisation: 16/11/2017,, , Revision: 19, Authorised, Last updated: 13/02/2023
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Human medicine European public assessment report (EPAR): Lutathera
lutetium (177Lu) oxodotreotide, Neuroendocrine Tumors
Date of authorisation: 26/09/2017,, Revision: 9, Authorised, Last updated: 13/02/2023
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Human medicine European public assessment report (EPAR): Tavneos
Avacopan, Microscopic Polyangiitis; Wegener Granulomatosis
Date of authorisation: 11/01/2022,, , Revision: 2, Authorised, Last updated: 13/02/2023
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Human medicine European public assessment report (EPAR): Sylvant
siltuximab, Giant Lymph Node Hyperplasia
Date of authorisation: 22/05/2014,, , Revision: 12, Authorised, Last updated: 09/02/2023
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Human medicine European public assessment report (EPAR): Defitelio
defibrotide, Hepatic Veno-Occlusive Disease
Date of authorisation: 18/10/2013,, , , Revision: 14, Authorised, Last updated: 09/02/2023
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Human medicine European public assessment report (EPAR): Fintepla
Fenfluramine hydrochloride, Epilepsies, Myoclonic
Date of authorisation: 18/12/2020,, , Revision: 4, Authorised, Last updated: 09/02/2023
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Human medicine European public assessment report (EPAR): Evrysdi
Risdiplam, Muscular Atrophy, Spinal
Date of authorisation: 26/03/2021,, , Revision: 3, Authorised, Last updated: 06/02/2023
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Human medicine European public assessment report (EPAR): Ninlaro
ixazomib citrate, Multiple Myeloma
Date of authorisation: 21/11/2016,, , , Revision: 15, Authorised, Last updated: 01/02/2023