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 |