List of the latest marketing authorisations and orphan medicinal products designations

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.

Orphan drug regulatory processLearn more about the Orphan designation process in Europe

 

 

Recent 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 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

 

 
 

 

Latest orphan medicines 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Applications withdrawn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

 

 

 
 
 
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