List of the marketing authorisations
Detailed information on European orphan medicinal products designation applications is available on the EMA website. A full list of designated and authorised orphan medicinal products in Europe available at: ec.europa.eu.
Learn more about the Orphan designation process in Europe
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Marketing authorisations
See a list of orphan medicines currently authorised in the EU.
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 disease14/02/2022 Ngenla®
(somatrogon)Pfizer Europe MA EEIG Children and
adolescents with
growth hormone
deficiency14/02/2022 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 -
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Applications withdrawn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