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List of the marketing authorisations

  • Marketing authorisations

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

    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

     

  • Orphan drug designations

    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    Applications withdrawn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