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2021 marketing authorisations

See a list of orphan medicines currently authorised in the EU.

Elzonris®

(tagraxofusp)

Stemline Therapeutics B.V. Adults with blastic
plasmacytoid dendritic
cell neoplasm
(BPDCN)
07/01/2021
Inrebic®
(fedratinib)
Celgene Europe BV Adults with myelofibrosis (a rare form of blood cancer) 08/02/2021
Lumoxiti®
(moxetumomab pasudotox)
AstraZeneca AB Adults with hairy cell leukaemia, a cancer of the white blood cells 08/02/2021
Sogroya®
(somapacitan)
Novo Nordisk A/S Growth hormone
deficiency
31/03/2021
Enspryng®
(satralizumab)
Roche Registration GmbH Neuromyelitis Optica
Spectrum disorders
(NMOSD)
24/06/2021
Bylvay®
(odevixibat)
Albireo +6 months with progressive familial intrahepatic cholestasis (PFIC) 16/07/2021
Skysona®
(elivaldogene autotemcel)
bluebird bio (Netherlands) B.V. Under 18 years of age with early cerebral adrenoleukodystrophy (CALD) 16/07/2021
Imcivree®
(setmelanotide)
Rhythm Pharmaceuticals Limited +6 years who have pro-opiomelanocortin (POMC) deficiency or leptin receptor (LEPR) deficiency 16/07/2021
Abecma®
(idecabtagene vicleucel)
Celgene Europe BV Adults with multiple myeloma (a cancer of the bone marrow) 18/08/2021
Voxzogo®
(vosoritide)
BioMarin International Limited Achondroplasia in patients aged +2 years 26/08/2021
Minjuvi®
(tafasitamab)
Incyte Biosciences Distribution B.V. Adults with diffuse large B-cell lymphoma (DLBCL) 26/08/2021
Qinlock®
(ripretinib)
Deciphera Pharmaceuticals
(Netherlands) B.V.
Gastrointestinal
Stromal Tumors
18/11/2021
Artesunate
Amivas
®
(artesunate)
Amivas Ireland Ltd Malaria 22/11/2021
Aspaveli®
(pegcetacoplan)
Swedish Orphan Biovitrum AB
(publ)
Adults with
paroxysmal nocturnal
haemoglobinuria
(PNH)
13/12/2021