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

2017 marketing authorisations

Medicinal Product

Marketing Authorisation Holder

Therapeutic Indication

Date of Marketing Authorisation

Alofisel®
(darvadstrocel)
Tigenix, S.A.U. Complex anal fistulas in adults with Crohn’s disease 23/03/2018
Amglidia®
(glibenclamide)
Ammtrek Neonatal diabetes 24/05/2018
Cablivic®
(caplacizumab)
Ablynx NV Acquired thrombotic thrombocytopenic purpura (aTTP) 31/10/2018
Crysvita®
(burosumab)
Kyowa Kirin Limited  X-linked hypophosphataemia  19/02/2018

Jorveza®
(budesonide)

Dr. Falk Pharma GmbH Eosinophilic oesophagitis 08/01/2018

Lamzede®
(velmanase alfa)

Chiesi Farmaceutici S.p.A. Alpha-mannosidosis 23/03/2018
Mepsevii®
(vestronidase alfametreleptin)
Ultragenyx Germany GmbH Mucopolysaccharidosis type VII (MPS VII, also known as Sly syndrome) 23/08/2018
Myalepta®
(metreleptin)
Aegerion Pharmaceuticals B.V. Lipodystrophy 30/07/2018

Mylotarg®
(gemtuzumab ozogamicin)

Pfizer Limited Acute myeloid leukemia 19/04/2018
Premyvis®
(letermovir)

Merck Sharp & Dohme Limited

Cytomegalovirus infection prevention following haematopoietic stem cell transplant 08/01/2018

Rubraca®
(rucaparib)

Clovis Oncology UK Limited High-grade cancers of the ovary, fallopian tubes and peritoneum

24/05/2018

 

Tegsedi®
(inotersen sodium)
Ionis USA Ltd Hereditary transthyretin amyloidosis (hATTR) 06/07/2018
Verkazia®
(ciclosporin)
Santen Oy Keratoconjunctivitis (VKC) in children and adolescents from 4 to 18 years of age. 06/07/2018

Yescarta®
(axicabtagene ciloleucel)

Kite Pharma EU B.V.  Diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL) 23/08/2018

 

Marketing authorisations since 2000

 
 

 

Latest orphan medicines designations

2018

2017

Overview of Orphan Medicinal Product Designation Procedure since 2000

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Applications withdrawn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

173

109

61

2

115

11

14

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

3148

2957

850

27

2067

153

171

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

 

 

 
 
 
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