Eurostat

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Absorption, Lower respiratory tract infection, Exercise, Cell, Infection, DSM-IV codes, Inflammation, Patient, Committee, Cystic fibrosis, Knowledge, Digestion, Regulation, Bacteria, Food, EMA, IRIS, Anti-inflammatory, Mucus, CB2, European, COMP, Pancreas, Sponsor, Ivacaftor, European Commission, Safety, CFTR, Marketing, Medicine, Pharmaceutical industry

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Key Points: 


Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Brain, Disease, Coagulation, Patient, Committee, Knowledge, Internal, Regulation, Haemophilia, EMA, IRIS, Joint, Human, Wound healing, Clinical trial, European, Therapy, COMP, Bleeding, Spinal cord, Sponsor, European Commission, Immune system, Antibody, Safety, Factor IX, Blood, Marketing, Limited, Haemophilia B, Medicine, Pharmaceutical industry

Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Key Points: 


Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Orphan designation: antisense oligonucleotide targeting exon 73 in the COL7A1 gene Treatment of epidermolysis bullosa, 12/10/2017 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Gene, Mutation, Epidermolysis bullosa, Skin cancer, Patient, Committee, Knowledge, Regulation, Keratin, EMA, IRIS, Protein, Life expectancy, Deformity, Blister, European, Skin, Collagen, COMP, Infection, Sponsor, European Commission, Safety, Unit, Marketing, COL7A1, Medicine, Pharmaceutical industry

Orphan designation: antisense oligonucleotide targeting exon 73 in the COL7A1 gene Treatment of epidermolysis bullosa, 12/10/2017 Withdrawn

Key Points: 


Orphan designation: antisense oligonucleotide targeting exon 73 in the COL7A1 gene Treatment of epidermolysis bullosa, 12/10/2017 Withdrawn

Orphan designation: branaplam Treatment of spinal muscular atrophy, 16/04/2018 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, SMN, Brain, Atrophy, Gene, Weakness, Spinal muscular atrophy, Survival, Medicine, Disease, Paralysis, Novartis, Patient, Committee, Knowledge, Ventilation, Regulation, Brain cell, EMA, IRIS, Mouth, European, COMP, Spinal cord, Sponsor, European Commission, Spine, Safety, SMN1, Marketing, SMN2, Pharmaceutical industry

Orphan designation: branaplam Treatment of spinal muscular atrophy, 16/04/2018 Withdrawn

Key Points: 


Orphan designation: branaplam Treatment of spinal muscular atrophy, 16/04/2018 Withdrawn

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Atrophy, Brain, Enzyme, DSM-IV codes, Cell, Patient, Committee, Knowledge, Mitochondrion, PPAR, Regulation, Fatigue, EMA, IRIS, Eye, Diet, European, Retina, COMP, Heart, Sponsor, European Commission, Hypoglycemia, Safety, LCHAD, Dicarboxylic acid, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn

Key Points: 


Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawn

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Mitochondrial encephalomyopathy, Headache, DSM-IV codes, Lactic acidosis, Weakness, MELAS, Cell, Disease, Seizure, Paratriathlon, Patient, Committee, Knowledge, Mitochondrion, Regulation, EMA, IRIS, European, COMP, Sponsor, European Commission, Safety, Muscle weakness, Pain, Vomiting, Marketing, Movement, Medicine, Pharmaceutical industry

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawn

Key Points: 


Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawn

Orphan designation: Tranilast Prevention of scarring post glaucoma filtration surgery, 27/07/2010 Positive

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Optic nerve, Brain, Pressure, Cell, Disease, Risk, Patient, Committee, Knowledge, Regulation, Cancer, EMA, IRIS, Scar, Eye, Wound healing, European, Allergic conjunctivitis, COMP, Collagen, Skin, European Commission, Safety, Glaucoma, Sclera, Pharmaceutical industry

Orphan designation: Tranilast Prevention of scarring post glaucoma filtration surgery, 27/07/2010 Positive

Key Points: 


Orphan designation: Tranilast Prevention of scarring post glaucoma filtration surgery, 27/07/2010 Positive

Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positive

Retrieved on: 
Thursday, April 18, 2024
Community, Eurostat, Civil service commission, Diagnosis, CD7, Graft-versus-host disease, Cell, Bone marrow, Disease, Patient, Committee, Extrapyramidal system, Tissue, Knowledge, Antibody, Regulation, EMA, IRIS, Protein, CD3, BV, European, Skin, WT1, COMP, Oxygen, Immune system, European Commission, Safety, Hematopoietic stem cell transplantation, Stomach, Liver, Pharmaceutical industry

Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positive

Key Points: 


Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positive

Orphan designation: N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide Treatment of neurofibromatosis type 1, 25/07/2019 Positive

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Optic nerve, Brain, Enzyme, Growth, Cell, NF1, Disease, Nervous system, Risk, Groin, Patient, Committee, Knowledge, Neoplasm, Regulation, Cancer, EMA, IRIS, Eye, European, Therapy, COMP, Safety, European Commission, Marketing, Medicine, Pharmaceutical industry

Orphan designation: N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide Treatment of neurofibromatosis type 1, 25/07/2019 Positive

Key Points: 


Orphan designation: N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide Treatment of neurofibromatosis type 1, 25/07/2019 Positive

Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positive

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, X-ray, Growth, Medicine, Disease, Patient, Ageing, Committee, Knowledge, Tissue, Neoplasm, Light, Cutaneous T-cell lymphoma, Regulation, Cancer, EMA, IRIS, Quality of life, Reactive oxygen species, Lymph, CTCL, Clinical trial, European, Skin, COMP, Oxygen, European Commission, Immune system, Safety, Carmustine, Alfa, Marketing, Lymphatic system, Pharmaceutical industry

Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positive

Key Points: 


Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positive