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January 2013 Orphan Drug Designations In Europe

February 6, 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 January 2013 Orphan Drug Designations in the EC is from the EURORDIS website :

1)  Treatment of familial adenomatous polyposis – Eflornithine in combination with sulindac

2)  Treatment of growth hormone deficiency – Recombinant modified human growth hormone

3)  Treatment of 5q spinal muscular atrophy – Allogeneic motor neuron progenitor cells derived from human embryonic stem cells

4)  Treatment of Wilson’s disease – Choline tetrathiomolybdate

5)  Treatment of pancreatic cancer – Recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem cell antigen

6)  Treatment of beta-thalassaemia intermedia and major – Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene

7)  Treatment of ovarian cancer – Chimeric monoclonal antibody against claudin 6

8)  Treatment of glioma – 1,2:5,6-Dianhydrogalactitol

9)  Treatment of achondroplasia – Modified recombinant human C-type natriuretic peptide

10) Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) – Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene

11)  Treatment of systemic sclerosis – Terguride

12)  Treatment of retinitis pigmentosa – Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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