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Orphan designation: 2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate for: Treatment of myelofibrosis
Date of designation: 28/02/2020, Positive, Last updated: 05/01/2022 -
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Orphan designation: Adeno-associated viral vector serotype S3 encoding human alpha-galactosidase A cDNA for: Treatment of Fabry disease
Date of designation: 28/02/2020, Positive, Last updated: 05/05/2020 -
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Orphan designation: Adeno-associated virus serotype 8 containing the human RdCVF sequence and the human RdCVFL sequence for: Treatment of inherited retinal dystrophies
Date of designation: 28/02/2020, Positive, Last updated: 05/05/2020 -
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Orphan designation: Adeno-associated virus serotype rh74 containing the human micro-dystrophin gene for: Treatment of Duchenne muscular dystrophy
Date of designation: 28/02/2020, Positive, Last updated: 22/10/2020 -
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Orphan designation: artesunate for: Treatment of malaria
Date of designation: 28/02/2020, Positive, Last updated: 13/01/2022 -
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Orphan designation: sutimlimab for: Treatment of immune thrombocytopenia
Date of designation: 28/02/2020, Positive, Last updated: 06/05/2020 -
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Orphan designation: Sintilimab for: Treatment of peripheral T-cell lymphoma
Date of designation: 28/02/2020, Positive, Last updated: 06/05/2020 -
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Orphan designation: Reldesemtiv for: Treatment of amyotrophic lateral sclerosis
Date of designation: 28/02/2020, Positive, Last updated: 06/05/2020 -
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Orphan designation: Luspatercept for: Treatment of myelofibrosis
Date of designation: 28/02/2020, Positive, Last updated: 18/02/2021 -
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Orphan designation: Combination of three adeno-associated viral vectors of serotype 8 containing the 5'-, the body- and the 3'- coding sequences of human CEP290 fused to inteins for: Treatment of inherited retinal dystrophies
Date of designation: 28/02/2020, Positive, Last updated: 07/11/2022 -
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Orphan designation: Autologous skin equivalent graft composed of keratinocytes and fibroblasts genetically corrected by CRISPR/Cas9-mediated excision of mutation-carrying COL7A1 exon 80 for: Treatment of epidermolysis bullosa
Date of designation: 28/02/2020, Positive, Last updated: 11/11/2022 -
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Orphan designation: Autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor for B-cell maturation antigen for: Treatment of multiple myeloma
Date of designation: 28/02/2020, Positive, Last updated: 13/06/2022