Skip to content
Generic filters
Exact matches only
Search in title
Search in content
Search in excerpt

List of the marketing authorisations

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

 

Medicinal Product Marketing Authorisation Holder Therapeutic Indication Date of Marketing Authorisation
Spexotras®
(Trametinib dimethyl sulfoxidetinib)
Novartis Europharm Limited Glioma 05/01/2024
Rystiggo®
(Rozanolixizumab)
UCB Pharma Myasthenia Gravis 05/01/2024
Omjjara®
(Momelotinib)
GlaxoSmithKline Trading
Services Limited
Splenomegaly 21/01/2024
Skyclarys®
(Omaveloxolone)
Reata Ireland Limited Friedreich’s ataxia 09/02/2024
Casgevy®
(Exagamglogene autotemcel)
Vertex Pharmaceuticals (Ireland) Limited Beta- Tahalassemia, Anemia, Sickle Cell 09/02/2024
Voydeya®
(Danicopan)
Alexion Europe SAS Paroxysmal nocturnal haemoglobinuria (PNH) 19/04/2024
Zynyz®
(Exagamglogene autotemcel)
Incyte Biosciences Distribution B.V. Merkel cell carcinoma (MCC) 19/04/2024

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

 

Medicinal Product Marketing Authorisation Holder Therapeutic Indication Date of Marketing Authorisation
Hemgenix®
(etranacogene dezaparvovec)
CSL Behring Gmbh Haemophilia B 20/02/2023
Tibsovo®
(ivosidenib)
Les Laboratoires Servier Acute Myeloid Leukaemia and cholangiocarcinoma 12/05/2023
Pombiliti®
(cipaglucosidase alfa)
Amicus Therapeutics Europe Limited Glycogen Storage Disease Type II 16/05/2023
Hyftor®
(sirolimus)
Plusultra pharma GmbH Facial angiofibroma 15/05/2023
Ztalmy®
(ganaxolone)
Marinus Pharmaceuticals Emerald Limited Epileptic seizures in children with CDKL5 26/07/2023
Talvey®
(talquetamab)
Janssen-Cilag International N.V. Multiple Myeloma 21/08/2023
Tepkinly®
(epcoritamab)
AbbVie Deutschland GmbH & Co. KG Diffuse large B-cell lymphoma (DLBCL) 22/09/2023
Finlee®
(dabrafenib esylate)
Novartis Europharm Limited Glioma 15/11/2023
Yorvipath®
(palopegteriparatide)
Ascendis Pharma Bone Diseases A/S Chronic hypoparathyroidism 17/11/2023
Loargys®
(pegzilarginase)
Immedica Pharma AB Hyperargininemia 15/12/2023
Agamree®
(vamorolone)
Santhera Pharmaceuticals (Deutschland) GmbH Duchenne muscular dystrophy 14/12/2023
Rezzayo®
(rezafungin)
Mundipharma GmbH Invasive candidiasis 22/12/2023

 

Medicinal Product Marketing Authorisation Holder Therapeutic Indication Date of Marketing Authorisation
Tavneos®
(avacopan)
Vifor Fresenius Medical Care Renal Pharma France Adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) 11/01/2022
Voraxaze®
(glucarpidase)
SERB SAS Adults and children (aged 28 days and older) with delayed methotrexate elimination or at risk of methotrexate toxicity 11/01/2022
Lonapegsomatropin Ascendis Pharma®
(lonapegsomatropin)
Ascendis Pharma Endocrinology Division A/S Children who do not produce enough growth hormone (GHD) 11/01/2022
Oxbryta®
(voxelotor)
Global Blood Therapeutics
Netherlands B. V.
Haemolytic anaemia,
and +12 years old
sickle cell disease
14/02/2022
Ngenla®
(somatrogon)
Pfizer Europe MA EEIG Children and
adolescents with
growth hormone
deficiency
14/02/2022
Kimmtrak®
(tebentafusp)
Immunocore Ireland Limited Adult patients with unresectable or metastatic uveal melanoma 01/04/2022
Uplizna®
(inebilizumab)
Viela Bio Adults with neuromyelitis optica spectrum disorders (NMOSD) 25/04/2022
Carvykti®
(ciltacabtagene autoleucel)
Janssen-Cilag International NV Adults with multiple myeloma 25/05/2022
Lunsumio®
(mosunetuzumab)
Roche Registration GmbH Adults with follicular lymphoma 03/06/2022
Filsuvez®
(birch bark extract)
Amryt Pharmaceuticals DAC Adults and children aged 6 months or older with epidermolysis bullosa (EB). 21/06/2022
Kinpeygo®
(budesonide)
Calliditas Therapeutics AB Adults with primary immunoglobulin A nephropathy (IgAN) 15/07/2022
Zokinvy®
(lonafarnib)
EigerBio Europe Limited 12 months and older living with progeria and laminopathies 18/07/2022
Upstaza®
(eladocagene exuparvovec)
PTC Therapeutics International Limited Adults and children aged 18 months and older with severe aromatic L-amino acid decarboxylase (AADC) deficiency 18/07/2022
Vyvgart®
(efgartigimod alfa)
Argenx Adults with myasthenia gravis 10/08/2022
Roctavian®
(valoctocogene roxaparvovec)
BioMarin International Limited Haemophilia A 24/08/2022
Scemblix®
(asciminib)
Novartis Europharm Limited Chronic myeloid leukaemia (CML) 25/08/2022
Enjaymo®
(sutimlimab)
Genzyme Europe BV Haemolytic anaemia in
adults with cold
agglutinin disease
(CAD)
15/11/2022
Livtencity®
(maribavir)
Takeda Pharmaceuticals Cytomegalovirus (CMV) in adults 9/11/2022
Pyrukynd®
(mitapivat)
Agios Netherlands Adults with pyruvate kinase deficiency (PKD) 9/11/2022
Livmarli®
(maralixibat chloride)
Mirum Pharmaceuticals 2 months and older patients with cholestatic pruritis 9/11/2022
Mycapssa®
(octreotide)
Amryt Pharmaceuticals DAC Acromegalia 2/12/2022
Ebvallo®
(tabelecleucel)
Atara Biotherapeutics Ireland Limited Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) 16/12/2022

 

  • Orphan drug designations

    2023

    2022

    2021

    2020

    2019

    2018

    2017

    Overview of Orphan Medicinal Product Designation Procedure since 2000

    Including and prior to 2018 the EMA also published the detailed information summarised below:

    Year

    Applications submitted

    Positive COMP opinions

    Applications withdrawn

    Negative COMP opinions

    Designations granted by the Commission

    Orphan medicinal products authorised

    Orphan designations included in authorised therapeutic indication

    2018

    203

    152

    79

    3

    126

    12

    15

    2017

    260

    144

    96

    2

    138

    14

    15

    2016

    330

    220

    82

    2

    209

    14

    14

    2015

    258

    177

    94

    1

    190

    14

    21

    2014

    329

    196

    61

    2

    187

    15

    16

    2013

    201

    136

    60

    1

    136

    7

    8

    2012

    197

    139

    52

    1

    148

    10

    12

    2011

    166

    111

    45

    2

    107

    5

    5

    2010

    174

    123

    51

    2

    128

    4

    4

    2009

    164

    113

    23

    0

    106

    9

    9

    2008

    119

    86

    31

    1

    73

    6

    7

    2007

    125

    97

    19

    1

    98

    13

    13

    2006

    104

    81

    20

    2

    80

    9

    11

    2005

    118

    88

    30

    0

    88

    4

    4

    2004

    108

    75

    22

    4

    73

    6

    6

    2003

    87

    54

    41

    1

    55

    5

    5

    2002

    80

    43

    30

    2

    49

    4

    4

    2001

    83

    62

    27

    1

    64

    3

    3

    2000

    72

    26

    6

    0

    14

    0

    0

    Total

    3211

    2134

    881

    28

    2121

    164

    185

    Adapted from the July 2018 Committee for Orphan Medicinal Products (COMP) meeting report