28 results
Keyword Aglae Remove keyword
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Orphan designation: Aglatimagene besadenovec for: Treatment of glioma (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023Aglatimagene besadenovec Overview This … and have a poor prognosis. Aglatimagene besadenovec, also known as … Aglatimagene besadenovec … -
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National expert: Steinunn Agla Gunnarsdóttir, Danish Medicines Agency (updated)
- Declaration of interests - 79.82 KB | PDF
- Curriculum Vitae - 16.65 KB | PDF
Steinunn Agla Gunnarsdóttir … Steinunn Agla Gunnarsdóttir … Vitae PERSONAL INFORMATION Steinunn Agla Gunnarsdottir WORK EXPERIENCE September … -
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National expert: Mehmet Ungan, European Medicines Agency (updated)
- Declaration of interests - 79.92 KB | PDF
- Curriculum Vitae - 60.22 KB | PDF
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Referral: Bovilis BVD
Article 33
Status: European Commission final decision, opinion/position date: 17/04/2007, EC decision date: 29/06/2007, Last updated: 07/12/2011Medivet Suppliers Ltd 87c Aglandjias Avenue PO Box 20932 CY-1665 … -
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National expert: Eva Bermejo-Sanchez, Spanish Agency of Medicines and Medical Devices (updated)
- Declaration of interests - 79.82 KB | PDF
- Curriculum Vitae - 64.7 KB | PDF
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Referral: Nimesulide
nimesulide, Article 31 referrals
Status: European Commission final decision, opinion/position date: 23/06/2011, EC decision date: 20/01/2012, Last updated: 19/04/2012 -
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Orphan designation: Crofelemer for: Treatment of microvillus inclusion disease (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Autologous T cells transduced with a lentiviral vector expressing a chimeric antigen receptor against CLL-1 for: Treatment of acute myeloid leukaemia (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Epeleuton for: Treatment of sickle cell disease (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Velusetrag for: Treatment of intestinal pseudo-obstruction (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Inaxaplin for: Treatment of apolipoprotein L1-mediated kidney disease (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Refusal of orphan designation for the prevention of spaceflight-related radiation and microgravity (new)
Date of refusal of designation: 14/10/2022, Negative, Last updated: 23/01/2023 -
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Orphan designation: Adeno‐associated viral vector serotype 8 containing the 3' human otoferlin coding sequence, adeno‐associated viral vector serotype 8 containing the 5' human otoferlin coding sequence for: Treatment of otoferlin gene-mediated hearing loss (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Eculizumab for: Treatment of Guillain-Barré syndrome (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Adeno-associated viral vector serotype rh.10 encoding the CLN2 gene for: Treatment of neuronal ceroid lipofuscinosis (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: isotretinoin for: Treatment of autosomal recessive congenital ichthyosis (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Human IgG4k monoclonal antibody against CD89 for: Treatment of linear IgA bullous dermatosis (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Etidronate disodium for: Treatment of pseudoxanthoma elasticum (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Sodium ({(2S)-1,4-bis[2-(4-chloro-3-fluorophenoxy)acetamido]bicyclo[2.2.2]octan-2-yl}oxy)methyl hydrogen phosphate-2-amino-2-(hydroxymethyl)propane-1,3-diol (1/1/1) for: Treatment of vanishing white matter disease (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Selinexor for: Treatment of myelofibrosis (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Methyl(R)-4-((3S,5R,7R,8R,9S,10S,13R,14S,17R)-7-hydroxy-10,13-dimethyl-3-((4-((pyridin-3-ylmethyl)amino)butyl)amino)hexadecahydro-1H-cyclopenta[a]phenanthren-17-yl)pentanoate for: Treatment of Rett syndrome (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Acetylleucine for: Treatment of GM1 gangliosidosis (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: ropeginterferon alfa-2b for: Treatment of essential thrombocythaemia (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Orphan designation: Oxygen, sodium chloride solution 0.9% for: Treatment of amyotrophic lateral sclerosis (new)
Date of designation: 11/10/2022, Positive, Last updated: 24/01/2023 -
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Committee for Orphan Medicinal Products (COMP): 6-8 September 2022
European Medicines Agency, Amsterdam, the Netherlands, from 06/09/2022 to 08/09/2022, Last updated: 21/10/2022