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December 2012 Orphan Drug Designations In Europe

January 18, 2013

A positive opinion on orphan designation is given by the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) based in London, with the final decision made by the European Commission (EC). The COMP was created as part of the EU Regulation on Orphan Medicinal Products to review EU applications from persons or companies for the purpose of seeking orphan medicinal product designations.

The following list of December 2012 Orphan Drug Designations in the EC is from the EURORDIS website :

1)  Treatment of lead toxicity – Erdosteine

2)  Treatment of malignant mesothelioma – Maytansinoid-conjugated human monoclonal antibody against mesothelin

3)  Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated) – Alisertib

4)  Treatment of acromegaly – Cyclo(-gamma-aminobutyryl-L-phenylalanyl-L-tryptophanyl-Dtryptophanyl-
L-lysyl-L-threonyl-L phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt

5)  Treatment of perinatal asphyxia – Allopurinol sodium

6)  Treatment of Duchenne muscular dystrophy – Exon 52 specific phosphorothioate oligonucleotide

7)  Treatment of Duchenne muscular dystrophy – Exon 55 specific phosphorothioate oligonucleotide

8)  Treatment of malaria – Artesunate

9)  Treatment of chronic lymphocytic leukaemia – 4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide

10)  Treatment of very-long-chain-acyl-CoA dehydrogenase deficiency – Triheptanoin

11)   Treatment of long-chain L-3-hydroxyacyl-CoA-dehydrogenase deficiency – Triheptanoin

12)   Treatment of chronic lymphocytic leukaemia – Humanised single chain monoclonal antibody against CD37

13)   Treatment of non-infectious uveitis – Voclosporin

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