The seventy-fifth meeting of the Committee for Orphan Medicinal Products (COMP) took place on 9-10 January 2007. The Committee was delighted to officially welcome two COMP members from the new Member States. An updated list of COMP membership is provided in Annex 1 (below).

COMP Opinions

The Committee adopted 6 positive opinions on orphan medicinal product designation during this meeting:

– Artesunate, from ACE Pharmaceuticals BV, for treatment of malaria (review time: day 62)

– Autologous dendritic cells pulsed with autologous cell lysate, from Dorian Regulatory Affairs BV, for treatment of glioma (review time: day 62)

– Ex-vivo cultured adult human mesenchymal stem cells, from Quintiles UK Limited, for treatment of Graft-versus-Host disease (review time: day 62)

– HLA class I/II binding tumour associated peptides (ADF-APO-CCN-GUC-K67-MET-MMP-MUC-RGS), from Immatics Biotechnologies GmbH, for treatment of renal cell carcinoma (review time: day 62)

– Idebenone, from Santhera Pharmaceuticals (Deutschland) AG, for treatment of Leber’s hereditary optic neuropathy (review time: day 90)

– Recombinant human C1-inhibitor, from Pharming Group N.V., for prevention of delayed graft function after solid organ transplantation (review time: day 90)

Four oral explanations on orphan medicinal product designation took place during the meeting.

Withdrawals of Orphan Medicinal Product Applications

The COMP noted that four applications for orphan medicinal product designation were withdrawn by the sponsor during evaluation phase of the procedure.

Overview of orphan designation procedures

The European Commission granted four positive decisions on orphan designation1 since the last COMP meeting on 5-6 December 2006 (see Annex 2).

The status of orphan designation procedures, to date for 2007, is summarised below:

– Applications submitted: 0

– Positive COMP Opinions: 6

– Applications Withdrawn: 3

– Appeals Ongoing: 0

– Final Negative COMP Opinions: 0

– Designations Granted by Commission: 0

An overview of orphan designation procedures for 2000-2006 is provided in Annex 3.

Further information on designated orphan medicinal products is publicly available in the form of summarised COMP Opinions2, which the Agency routinely publishes following adoption of the respective decisions on orphan designation by the European Commission.

Applications for Marketing Authorisation for Orphan Medicinal Products

Details of those designated orphan medicinal products that have been the subject of a centralised application for marketing authorisation since the last COMP meeting are provided in Annex 4.

Date of next COMP meeting

The next COMP meeting will be held on 6-7 February 2007.

1. Details of all orphan designations granted to date by the European Commission are entered in the Community Register of Orphan Medicinal Products (link here)

ANNEX 1 to COMP January 2007
Composition of the Committee for Orphan Medicinal Products

Chair: Kerstin Westermark
EMEA contact: Agn??s SAINT RAYMOND

Members
??? Brigitte BL-CHL-DAUM (Austria)
??? Andrew BORG (Malta)
??? Heidrun BOSCH TRABERG (Denmark)
??? Birthe BYSKOV HOLM (patients’ organisation representative) (vice-chairman)
??? Yann LE CAM (patients’ organisation representative)
??? Ana CORR??A NUNES (Portugal)
??? Boz?‡enna DEMBOWSKA-BAGIN??SKA (Poland)
??? Julia DUNNE (EMEA representative)
??? Judit EGGENHOFER (Hungary)
??? Rembert ELBERS (Germany)
??? Pauline EVERS (patients’ organisation representative)
??? Lars GRAMSTAD (Norway)
??? Emmanuel H?‰RON (France)
??? Detelina IVANOVA (Bulgaria)
??? Ioannis KKOLOS (Cyprus)
??? Kater??ina KUBA??C??KOV?? (Czech Republic)
??? Magdal?©na KUZ??ELOV?? (Slovakia)
??? Andr?© LHOIR (Belgium)
??? David LYONS (EMEA representative)
??? Greg MARKEY (United Kingdom)
??? Aus??ra MATULEVIC??IENE?‡ (Lithuania)
??? Henri METZ (Luxembourg)
??? Martin MOZ??INA (Slovenia)
??? Kristina PAVLOVSKA (Latvia)
??? Veijo SAANO (Finland)
??? Patrick SALMON (Ireland)
??? Miranda SIOUTI (Greece)
??? Daniela STANCIU (Romania)

ANNEX 2 to COMP January 2007

Orphan Medicinal Product Designations received since the December 2006 COMP Meeting

Active substance – 4,7,10,13,16,19-docosahexaenoic acid
Sponsor – Jose Manuel Cela Lopez
Orphan Indication – Treatment of retinitis pigmentosa
Opinion receipt date – 17 October 2006
Date of Commission Decision – 3 November 2006

Active substance – Forodesine hydrochloride
Sponsor – Napp Pharmaceuticals Research Limited
Orphan Indication – Treatment of acute lymphoblastic leukaemia
Opinion receipt date – 20 November 2006
Date of Commission Decision – 18 December 2006

Active substance – Paclitaxel (micellar)
Sponsor – Oasmia Pharmaceutical AB
Orphan Indication – Treatment of ovarian cancer
Opinion receipt date – 20 November 2006
Date of Commission Decision – 18 December 2006

Active substance – Tazarotene
Sponsor – Orfagen
Orphan Indication – Treatment of congenital ichthyoses
Opinion receipt date – 20 November 2006
Date of Commission Decision – 18 December 2006

ANNEX 3 to COMP January 2007
Overview of Procedures for Orphan Medicinal Product Designation for 2000-2006

2006
Applications submitted – 104
Positive COMP Opinions – 81
Applications withdrawn – 20
Final negative COMP Opinions – 2
Designations granted by Commission – 80

2005
Applications submitted – 118
Positive COMP Opinions – 88
Applications withdrawn – 30
Final negative COMP Opinions – 0
Designations granted by Commission – 88

2004
Applications submitted – 108
Positive COMP Opinions – 75
Applications withdrawn – 22
Final negative COMP Opinions – 4
Designations granted by Commission – 72

2003
Applications submitted – 87
Positive COMP Opinions – 54
Applications withdrawn – 41
Final negative COMP Opinions – 1
Designations granted by Commission – 55

2002
Applications submitted – 80
Positive COMP Opinions – 43
Applications withdrawn – 30
Final negative COMP Opinions – 3
Designations granted by Commission – 49

2001
Applications submitted – 83
Positive COMP Opinions – 64
Applications withdrawn – 27
Final negative COMP Opinions – 1
Designations granted by Commission – 64

2000
Applications submitted – 72
Positive COMP Opinions – 26
Applications withdrawn – 6
Final negative COMP Opinions – 0
Designations granted by Commission – 14

ANNEX 4 to COMP January 2007
Overview of Designated Orphan Medicinal Products that have been the subject of a Centralised Application for Marketing Authorisation

- update since the last COMP meeting on 6-7December 2006-

Active substance
gusperimus trihydrochloride (Spanidin)
Sponsor/applicant
Euro Nippon Kayaku GmbH
EU Designation Number & Date of Orphan Designation
EU/3/01/034 29/03/2001
Designated Orphan Indication
Treatment of Wegener’s granulomatosis

emea.eu.int