Membership list: HMA-EMA joint Big Data Steering Group

Orphan designation: Autologous T lymphocytes containing a T-cell receptor against Epstein–Barr virus Treatment of peripheral T-cell lymphoma, 13/12/2023 Positive

Orphan designation: 2'-O, 4'-C-Methylene-P-thio-adenylyl-(3'->5')-2'-O, 4'-C-methylene-P-thioguanylyl-(3'->5')-2'-O, 4'-C-methylene-P-thio-adenylyl-(3'->5')-2'-deoxy-P-thioadenylyl-(3'->5')-2'-deoxy-P-thio-thymidylyl-(3'->5')-2'-deoxy-P-thio-guanylyl-(3'->5')-2'-deoxy-P-thio-guanylyl-(3'->5')-2'-deoxy-P-thio-cytidylyl-(3'->5')-2'-deoxy-P-thio-adenylyl-(3'->5')-2'-deoxy-P-thio-cytidylyl-(3'->5')-2'-deoxy-P-thio-adenylyl-(3'->5')-2'-deoxy-P-thio-thymidylyl-(3'->5')-2'-deoxy-P-thio-cytidylyl-(3'->5')-2'-deoxy-P-thio-thymidylyl-(3'->5')-2'-O, 4'-C-methy

Orphan designation: brogidirsen Treatment of Duchenne muscular dystrophy, 13/12/2023 Positive

Orphan designation: Humanised IgG1 kappa fragment antibody targeting TfR1 conjugated to P125 oligonucleotide Treatment of myotonic disorders, 22/05/2023 Positive

Orphan designation: Govorestat Treatment of Charcot-Marie-Tooth disease, 22/05/2023 Positive

Orphan designation: 2-((2-fluoro-4-iodophenyl)amino)-N-(2-hydroxyethoxy)-1-methyl-1H-pyrrolo[2,3-b]pyridine-3-carboxamide Treatment of neurofibromatosis type 1, 22/05/2023 Positive

Orphan designation: Fingolimod Treatment of hypomyelinating leukodystrophy-18, 22/05/2023 Positive

Orphan designation: Efzofitimod Treatment of systemic sclerosis, 20/06/2023 Positive

Orphan designation: Fasudil hydrochloride Treatment of amyotrophic lateral sclerosis, 20/06/2023 Positive