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33 results
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Orphan designation: arimoclomol citrate for: Treatment of inclusion body myositis
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Arimoclomol for: Treatment of amyotrophic lateral sclerosis
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Moxetumomab pasudotox for: Treatment of B-lymphoblastic leukaemia/lymphoma
Date of designation: 19/07/2022, Withdrawn, Last updated: 25/07/2022 -
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Orphan designation: Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1 (naratuximab emtansine) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Teclistamab for: Treatment of multiple myeloma
Date of designation: 19/07/2022, Withdrawn, Last updated: 13/10/2022 -
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Orphan designation: Odronextamab for: Treatment of diffuse large B-cell lymphoma
Date of designation: 18/07/2022, Positive, Last updated: 25/01/2023 -
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Orphan designation: Salmonella enterica, subsp. enterica, serovar Typhimurium, strain YS1646, live for: Treatment of schwannoma
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Berzosertib for: Treatment of small cell lung cancer
Date of designation: 18/07/2022, Withdrawn, Last updated: 07/02/2023 -
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Orphan designation: Heterologous swine glyco-humanised polyclonal antibody against T lymphocytes for: Treatment in solid organ transplantation
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Parsaclisib for: Treatment of autoimmune haemolytic anaemia
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Panobinostat for: Treatment of glioma
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: zilucoplan for: Treatment of myasthenia gravis
Date of designation: 18/07/2022, Positive, Last updated: 25/01/2023 -
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Orphan designation: Efgartigimod alfa for: Treatment of pemphigus
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Vilobelimab for: Treatment of pyoderma gangrenosum
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Odronextamab for: Treatment of follicular lymphoma
Date of designation: 18/07/2022, Positive, Last updated: 25/01/2023 -
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Orphan designation: Sodium phenylbutyrate for: Treatment of maple syrup urine disease
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Thiostrepton for: Treatment of malignant mesothelioma
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: (S)-1-(4-(1-(3,4,5-trimethoxyphenyl)-1H-imidazol-4-ylamino)thieno[2,3-d]pyrimidin-2-yl)pyrrolidine-2-carboxamide for: Treatment of fibrodysplasia ossificans progressiva
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Human decorin fused to the truncated homing peptide CRK for: Treatment of epidermolysis bullosa
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: 3-(1,3-benzodioxol-5-yl)-5-(3-bromophenyl)-1H-pyrazole for: Treatment of multiple system atrophy
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Pyridoxal 5'-phosphate for: Treatment of pyridoxal 5'‐phosphate homeostasis protein deficiency
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Hydroquinidine hydrochloride for: Treatment of Brugada syndrome
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: Autologous naive regulatory T cells transduced with a lentiviral vector encoding for a chimeric antigen receptor to recognise the HLA-A*02 antigen for: Treatment in solid organ transplantation
Date of designation: 18/07/2022, Positive, Last updated: 25/01/2023 -
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Orphan designation: W253R/R275S tissue plasminogen activator for: Treatment of non-traumatic spontaneous intracerebral haemorrhage
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023 -
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Orphan designation: mRNA encoding modified human ornithine transcarbamylase for: Treatment of ornithine transcarbamylase deficiency
Date of designation: 18/07/2022, Positive, Last updated: 23/01/2023