2020 marketing authorisations

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

Medicinal Product

Marketing Authorisation Holder

Therapeutic Indication

Date of Marketing Authorisation

Isturisa®
(osilodrostat)
Novartis Europharm Limited Cushing Syndrome 09/01/2020
Polivy®
(polatuzumab vedotin)
Roche Registration GmbH Diffuse large B-cell lymphoma (DLBCL) 16/01/2020
Givlaari®
(givosiran)
Alnylam Netherlands B.V. Acute hepatic
porphyria
02/03/2020
Trepulmix®
(treprostinil)
SciPharm Sàrl Chronic
thromboembolic
pulmonary
hypertension
(CTEPH)
03/04/2020
Zolgensma®
(onasemnogene
abeparvovec)
AveXis EU Limited Spinal muscular
atrophy (SMA)
18/05/2020
Reblozyl®
(luspatercept)
Celgene Europe B.V. Beta thalassaemia & Myelodysplastic syndromes 25/06/2020
Daurismo®
(glasdegib)
Pfizer Europe MA EEIG Newly-diagnosed acute myeloid leukaemia (AML) 26/06/2020
Pretomanid FGK
(pretomanid)
FGK Representative Service GmbH Adults with drug-resistant tuberculosis 31/07/2020
Hepcludex
(bulevirtide)
MYR GmbH Chronic (long-term) hepatitis delta virus (HDV) infection in adults 31/07/2020
Idefirix
(imlifidase)
Hansa Biopharma AB Prevent the body from rejecting a newly transplanted kidney 25/08/2020
Kaftrio
(ivacaftor / tezacaftor / elexacaftor)
Vertex Pharmaceuticals (Ireland) Limited Cystic fibrosis 21/08/2020
Blenrep
(belantamab mafodotin)
GlaxoSmithKline (Ireland) Limited Multiple Myeloma 25/08/2020
Ayvakyt
(avapritinib)
Blueprint Medicines (Netherlands) B.V. Gastrointestinal stromal tumour (GIST) 24/09/2020
Arikayce liposomal
(amikacin sulfate)
Insmed Netherlands B.V. Adults with a lung
infection caused by
Mycobacterium avium
complex (MAC)
27/10/2020
Adakveo
(crizanlizumab)
Novartis Europharm Limited Anemia, Sickle Cell 28/10/2020
Obiltoxaximab SFL
(obiltoxaximab)
SFL Pharmaceuticals Deutschland GmbH Anthrax 18/11/2020
Oxlumo
(lumasiran)
Alnylam Netherlands B.V. Hyperoxaluria, Primary 19/11/2020
Libmeldy
(autologous CD34+
cells encoding ARSA
gene)
Orchard Therapeutics
(Netherlands) BV
Metachromatic
leukodystrophy (MLD)
17/12/2020
Fintepla®
(fenfluramine)
Zogenix ROI Limited Seizures associated
with Dravet syndrome
18/12/2020

 

Page created: 21/02/2021
Page last updated: 21/02/2021
 
 
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