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

Medicinal Product

Marketing Authorisation Holder

Therapeutic Indication

Date of Marketing Authorisation

Alprolix®
(eftrenacog alfa)

Biogen Idec Ltd

Haemophilia B

12/05/2016

Coagadex®
(human coagulation factor X)

Bio Products Laboratory Limited

Hereditary factor X deficiency

16/03/2016

Darzalex®
(daratumumab)

Janssen-Cilag International N.V.

Multiple myeloma

20/05/2016

Galafold®
(migalastat)

Amicus Therapeutics UK Ltd

Fabry disease

16/05/2016

Idelvion®
(albutrepenonacog alfa)

CSL Behring GmbH

Haemophilia B

11/05/2016

Strimvelis®
(autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence)

GlaxoSmithKline Trading Services Limited

Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)

26/05/2016

Uptravi®
(selexipag)*

Actelion Registration Ltd

Pulmonary arterial hypertension

12/05/2016

Wakix®
(pitolisant)

Bioprojet Pharma

Narcolepsy

31/03/2016

 

*Removed from the Community register of orphan medicinal products upon request of the MAH at the time of marketing authorisation

Marketing authorisations since 2000

 
 

 

Latest orphan medicines designations

 

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Applications withdrawn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

2016

173

128

35

0

112

7

7

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

2558

1735

642

21

1708

121

135

Adapted from the July 2016 Committee for Orphan Medicinal Products (COMP) meeting report

 

 
 
 
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