Orphans: Regulatory and procedural guidance and forms
Submitted by Anonymous (not verified) on 19 January 2024 - 15:38
Orphans: Regulatory and procedural guidance and forms
Third Veterinary Big Data Stakeholder Forum, European Medicines Agency, Amsterdam, the Netherlands, 23 November 2023
Submitted by Anonymous (not verified) on 19 January 2024 - 14:21
Third Veterinary Big Data Stakeholder Forum, European Medicines Agency, Amsterdam, the Netherlands, 23 November 2023
Orphan designation: Bitopertin Treatment of erythropoietic protoporphyria, 15/02/2023 Positive
Submitted by Anonymous (not verified) on 19 January 2024 - 13:54
Orphan designation: Bitopertin Treatment of erythropoietic protoporphyria, 15/02/2023 Positive
Orphan designation: (2S,3R,4R,5S)-1-[5-(2-Fluoro-biphenyl-4-ylmethoxy)-pentyl]-2-hydroxymethyl-piperidine-3,4,5-triol Treatment of GM2 gangliosidosis, 15/02/2023 Positive
Submitted by Anonymous (not verified) on 19 January 2024 - 13:49
Orphan designation: (2S,3R,4R,5S)-1-[5-(2-Fluoro-biphenyl-4-ylmethoxy)-pentyl]-2-hydroxymethyl-piperidine-3,4,5-triol Treatment of GM2 gangliosidosis, 15/02/2023 Positive
Orphan designation: autologous CD34+ cells transduced with a lentiviral vector containing the human GAA gene Treatment of glycogen storage disease type II (Pompe's disease), 15/02/2024 Positive
Submitted by Anonymous (not verified) on 19 January 2024 - 13:36
Orphan designation: autologous CD34+ cells transduced with a lentiviral vector containing the human GAA gene Treatment of glycogen storage disease type II (Pompe's disease), 15/02/2024 Positive
Orphan designation: crisdesalazine Treatment of amyotrophic lateral sclerosis, 15/02/2023 Positive
Submitted by Anonymous (not verified) on 19 January 2024 - 11:44
Orphan designation: crisdesalazine Treatment of amyotrophic lateral sclerosis, 15/02/2023 Positive
Orphan designation: humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavable linker treatment of facioscapulohumeral muscular dystrophy, 15/02/2023 Positive
Submitted by Anonymous (not verified) on 19 January 2024 - 11:37
Orphan designation: humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavable linker treatment of facioscapulohumeral muscular dystrophy, 15/02/2023 Positive
Summary of opinion: Metacam, 17/01/2024 Positive
Submitted by Anonymous (not verified) on 19 January 2024 - 11:26
Summary of opinion: Metacam, 17/01/2024 Positive
Meeting highlights from the Committee for Veterinary Medicinal Products (CVMP) 16-17 January 2024
Submitted by Anonymous (not verified) on 19 January 2024 - 11:25
Meeting highlights from the Committee for Veterinary Medicinal Products (CVMP) 16-17 January 2024
Applying for orphan designation
Submitted by Anonymous (not verified) on 19 January 2024 - 10:48
Applying for orphan designation