EMA

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of Rett syndrome, 10/08/2015 Positive

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Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Speech, X chromosome, Brain, Medicine, MECP2, Disease, Laxative, Seizure, Nervous system, Patient, Chromosome, Committee, Knowledge, Constipation, Regulation, EMA, IRIS, Rett syndrome, Male, Intellectual disability, Clinical trial, European, COMP, Safety, European Commission, Marketing, Parent, Syndrome, DSM-IV codes, Anatomical pathology, Pharmaceutical industry

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of Rett syndrome, 10/08/2015 Positive

Key Points: 


Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of Rett syndrome, 10/08/2015 Positive

Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in ha[...]

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Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Graft-versus-host disease, Medicine, Cell, Bone marrow, Risk, Haematopoiesis, Patient, Committee, Knowledge, Regulation, HSCT, EMA, IRIS, European, Leukemia, COMP, Immune system, European Commission, Consultant, Safety, Haematopoietic system, Blood, Marketing, Umbilical cord, CATS, Pharmaceutical industry

Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in haematopoietic stem cell transplantation, 20/04/2020 Positive

Key Points: 


Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in haematopoietic stem cell transplantation, 20/04/2020 Positive

Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

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Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, WHIM, WHIM syndrome, Myelokathexis, Pneumonia, Bone marrow, CXCR4, Risk, Patient, Committee, Knowledge, Hypogammaglobulinemia, Antibody, Regulation, Viral, EMA, IRIS, Wart, Otitis, Protein, Lymphocyte, European, Infection, COMP, Immune system, European Commission, Neutrophil, Blood, Safety, Consultant, Marketing, Movement, Pharmaceutical industry

Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

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Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

Orphan designation: Azithromycin dihydrate Prevention of bronchopulmonary dysplasia, 14/01/2022 Positive

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Tuesday, April 9, 2024
Community, Diagnosis, Civil service commission, Bronchopulmonary dysplasia, Ureaplasma urealyticum infection, BPD, Prevalence, Protein, Disease, Infection, Clinical, Inflammation, Patient, Committee, Bacteria, EMA, IRIS, Update, Agency, European, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the prevention of bronchopulmonary dysplasia in the European Union on 14 January 2022.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Orphan designation: Allogeneic cultured postnatal thymus-derived tissue Treatment of CHARGE syndrome, 26/02/2019 Positive

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Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Gene, Brain, Growth, Coloboma, Thymus, Patient, Committee, Knowledge, CHD7, Regulation, Child, Ventricular septal defect, EMA, IRIS, Eye, European, Infection, Therapy, COMP, Heart, Safety, European Commission, Spine, CHARGE syndrome, Nose, Genetic disorder, Sleep, Hearing, Medicine, Pharmaceutical industry

Orphan designation: Allogeneic cultured postnatal thymus-derived tissue Treatment of CHARGE syndrome, 26/02/2019 Positive

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Orphan designation: Allogeneic cultured postnatal thymus-derived tissue Treatment of CHARGE syndrome, 26/02/2019 Positive

Orphan designation: Allogeneic cultured postnatal thymus-derived tissue Treatment of DiGeorge syndrome, 26/02/2019 Positive

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Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Learning, Vitamin D, Medicine, DiGeorge syndrome, Seizure, Patient, Committee, Knowledge, Regulation, Ventricular septal defect, Palate, EMA, IRIS, Mouth, European, Infection, COMP, Heart, Safety, European Commission, Anatomical pathology, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    On 26 February 2019, orphan designation (EU/3/19/2135) was granted by the European Commission to Enzyvant Therapeutics Ireland Limited, Ireland, for allogeneic cultured postnatal thymus-derived tissue (also known as RVT-802) for the treatment of DiGeorge syndrome.
  • DiGeorge syndrome is a long-term debilitating condition that may be life-threatening particularly because of the heart problems and serious infections.
  • At the time of application for orphan designation, there was no satisfactory treatment for DiGeorge syndrome authorised in the EU.
  • Orphan designation of the medicine had been granted in the United States for treatment of patients with complete DiGeorge anomaly.

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

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Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Brain, B cell, Gaucher's disease, Cell, CD34, Bone marrow, Bleeding, Disease, Seizure, Bruise, Patient, Committee, Knowledge, Fracture, Spleen, Regulation, Fatigue, Autotransplantation, EMA, IRIS, Human, Life expectancy, Eye, Bone pain, Anemia, European, COMP, Sponsor, European Commission, Safety, PPD, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

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Tuesday, April 9, 2024
Eurostat, Civil service commission, Diagnosis, Brain, Bleeding, Disease, Coagulation, Patient, Committee, Knowledge, Regulation, Haemophilia, EMA, IRIS, Joint, Protein, Human, Clinical trial, European, COMP, Spinal cord, Antibody, Factor VIII, Sponsor, European Commission, Immune system, Safety, Blood, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

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Tuesday, April 9, 2024
Diamond, Community, MC1R, Civil service commission, Narcolepsy, Diagnosis, Pigment, Erythropoietic protoporphyria, Prevalence, Disease, EPP, Patient, Committee, EMA, IRIS, Agency, European, Skin, COMP, Sponsor, European Commission, Pain, Phototoxicity, PAH, Marketing, Phenylalanine hydroxylase, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Key Points: 


Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

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Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Fabry disease, Brain, Cell, CD34, Disease, Nervous system, Patient, Committee, Knowledge, Tissue, Stroke, Kidney failure, Fats, Regulation, Autotransplantation, EMA, IRIS, Human, Eye, Sugar, European, Skin, COMP, Heart, Sponsor, European Commission, Enzyme replacement therapy, Blood, PPD, Pain, Safety, Marketing, Bone marrow, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn