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Europe’s Orphan Product Designations: 2013 In Review

January 15, 2014



Updated 01/16/14 for 2012 EMA COMP ODDs to 139.

This Blog Post will compare the number of EMA COMP’s (European Medicines Agency’s Committee for Orphan Medicinal Products) positive opinions recommending Orphan Drug Designation (ODD) in 2012 versus the number of EMA COMP ODDs in 2013. The source of the data for both years are the EMA COMP Meeting Reports that are generated each month.

EMA COMP 2012 and 2013 ODDs

# of 2012 EMA COMP   ODDs Granted

# of 2013 EMA   COMP ODDs Granted

%     Change From 2012 To 2013



– 2.15 %


Some of the ODDs granted in 2013 include:

•   Theradex (Europe) Ltd.’s Poloxamer 188 for Sickle Cell Disease

•   International Niemann-Pick Disease Alliance’s (INPDA) 2-hydroxypropyl-β-cyclodextrin for Niemann-Pick Disease Type C

•   Synageva BioPharma’s Recombinant human alpha-N-acetylglucosaminidase for Mucopolysaccharidosis Type IIIB (Sanfilippo B syndrome)

•   Celgene Europe Limited’s Apremilast for Behçet’s Disease.

The number of EMA COMP ODDs remains flat from 2012 to 2013 – there is a very slight  2.15% decrease. In 2013, the FDA almost doubles the number of ODDs compared to the number of EMA COMP ODDs. Below is a chart of the 2012 and 2013 FDA ODDs.

FDA 2012 and 2013 ODDs

# of 2012 FDA   ODDs Granted

# of 2013 FDA   ODDs Granted

%     Change From 2012 To 2013



35.3 %


Please Note: “Cambio de color a diferente pHs” by Rubashkyn (Own work) [CC-BY-SA-3.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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