Regulation

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

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Tuesday, April 9, 2024
Eurostat, Civil service commission, Wasting, Diagnosis, Atrophy, Mutation, B cell, Weakness, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Death, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, Safety, European Commission, Muscle weakness, Marketing, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Key Points: 


Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Orphan designation: Nirogacestat Treatment of soft tissue sarcoma, 17/10/2019 Positive

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Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Plantar fibromatosis, Radiation, Gene, Growth, Sarcoma, Blood, Disease, Patient, Committee, Knowledge, Tissue, Regulation, Cancer, EMA, IRIS, Radiation therapy, European, Therapy, COMP, Aggressive fibromatosis, Safety, European Commission, GS, Pain, Marketing, Swelling, Medicine, Pharmaceutical industry

Orphan designation: Nirogacestat Treatment of soft tissue sarcoma, 17/10/2019 Positive

Key Points: 


Orphan designation: Nirogacestat Treatment of soft tissue sarcoma, 17/10/2019 Positive

Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1[...]

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Tuesday, April 9, 2024
Eurostat, Diagnosis, Failure, Civil service commission, Enzyme, Protease, Disease, Infection, Inflammation, Patient, Committee, Knowledge, Netherton syndrome, Regulation, EMA, IRIS, Life, Protein, Kallikrein, European, Skin, COMP, Water, Safety, European Commission, MDC, Calcineurin, Moisturizer, Steroid, Syndrome, Medicine, Pharmaceutical industry

Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide} Treatment of Netherton syndrome, 17/10/2019 Positive

Key Points: 


Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide} Treatment of Netherton syndrome, 17/10/2019 Positive

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

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Tuesday, April 9, 2024
Eurostat, Fragile X syndrome, Civil service commission, Diagnosis, X chromosome, Brain, FMRP, Woman, Learning disability, Disease, Nervous system, Patient, Committee, Knowledge, History, Regulation, Traffic barrier, Anxiety, EMA, IRIS, Mouth, European, COMP, European Commission, Consultant, Safety, Marketing, DSM-IV codes, Turner syndrome, Medicine, Pharmaceutical industry

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

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Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

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

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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

Key Points: 


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

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