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
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
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
Meeting highlights from the Committee for Veterinary Medicinal Products (CVMP) 16-17 January 2024
Applying for orphan designation
European Medicines Agency (EMA) Patients' and Consumers' (PCWP) and Healthcare Professionals' (HCPWP) Working Parties meeting with all eligible organisations, European Medicines Agency, Amsterdam, the Netherlands, from 14 November 2023 to 15 November 2023
Recommended submission dates for veterinary medicinal products