Civil service commission

Orphan designation: Alisitol, retinol palmitate, zinc gluconate Treatment of microvillus inclusion disease, 13/11/2020 Positive

Retrieved on: 
Mardi, avril 9, 2024
Eurostat, Civil service commission, Salt, Diagnosis, Absorption, Microvillous inclusion disease, DSM-IV codes, Nutrient, Cell, Patient, Committee, Weight loss, Knowledge, Regulation, Food, EMA, IRIS, Noetics, Parenteral nutrition, Diarrhea, European, Malnutrition, COMP, Dehydration, Water, Safety, European Commission, Liver, Medicine, Pharmaceutical industry

Orphan designation: Alisitol, retinol palmitate, zinc gluconate Treatment of microvillus inclusion disease, 13/11/2020 Positive

Key Points: 


Orphan designation: Alisitol, retinol palmitate, zinc gluconate Treatment of microvillus inclusion disease, 13/11/2020 Positive

Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Positive

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Mardi, avril 9, 2024
Eurostat, Diagnosis, Civil service commission, Leber's hereditary optic neuropathy, Prevalence, Cell, Patient, Committee, Knowledge, Seriousness, Mitochondrion, Regulation, EMA, IRIS, Adolescence, LHON, European, COMP, Oxygen, Safety, European Commission, Marketing, Visual impairment, Medicine, Pharmaceutical industry

Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Positive

Key Points: 


Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Positive

Orphan designation: imatinib Treatment of pulmonary arterial hypertension, 21/06/2021 Positive

Retrieved on: 
Mardi, avril 9, 2024
Community, Diagnosis, Civil service commission, Enzyme, Prevalence, Cell, Disease, Patient, Committee, Cancer, EMA, IRIS, Imatinib, Orthostatic hypertension, Agency, European, COMP, European Commission, MDC, PAH, Marketing, FGK, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of pulmonary arterial hypertension (PAH) in the European Union on 21 June 2021.
  • 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: Autologous CD34+ cells transduced with a lentiviral RNA vector that results in integrated cDNA encoding for functional cystinosin Treatment of cystinosis, 19/02/2021 Positive

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Mardi, avril 9, 2024
Community, Diagnosis, Civil service commission, Prevalence, Cell, Bone marrow, Disease, Novartis, Patient, Committee, RNA, Autotransplantation, EMA, IRIS, Cystinosis, Agency, European, COMP, Blood, European Commission, Marketing, Medicine, Pharmaceutical industry

All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

Key Points: 
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The medicine, also known as AVR-RD-O4, is a gene therapy, produced from immature blood (stem) cells collected from the patient.
  • After infusion into the patient, these cells permanently settle in the bone marrow, where they multiply to produce mature blood cells.
  • 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: melatonin Treatment of perinatal asphyxia, 02/04/2012 Positive

Retrieved on: 
Mardi, avril 9, 2024
Eurostat, Diagnosis, Civil service commission, Research, Brain, Disability studies, Survival, Asphyxia, Patient, Committee, Knowledge, Regulation, Child, Melatonin, EMA, IRIS, Thermoregulation, Intellectual, European, COMP, Oxygen, Safety, European Commission, Placenta, Hypothermia, Blood, Umbilical cord, Marketing, Perinatal asphyxia, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    On 2 April 2012, orphan designation (EU/3/12/978) was granted by the European Commission to Dr Nicola J Robertson, United Kingdom, for melatonin for the treatment of perinatal asphyxia.
  • At the time of orphan designation, there was no treatment for perinatal asphyxia authorised in the EU.
  • Key facts
    - Active substance
    - melatonin
    - Intended use
    - Treatment of perinatal asphyxia
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/12/978
    - Date of designation
    - Sponsor
    UCL Research Limited
    Review of designation
    The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
  • 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: Olorofim Treatment of invasive Scopulariopsis, 14/01/2022 Positive

Retrieved on: 
Mardi, avril 9, 2024
Community, Diagnosis, Civil service commission, Growth, Prevalence, Survival, Disease, Clinical, Patient, Committee, EMA, IRIS, Update, Scopulariopsis, Agency, European, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of invasive Scopulariopsis 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: 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

Retrieved on: 
Mardi, avril 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: Adeno-associated virus serotype rh10 containing the human GALC gene Treatment of Krabbe disease, 15/10/2021 Positive

Retrieved on: 
Mardi, avril 9, 2024
Diamond, Community, Diagnosis, Civil service commission, Prevalence, Disease, Patient, Committee, EMA, IRIS, Krabbe disease, GALC, Agency, European, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of Krabbe disease in the European Union on 15 October 2021.
  • 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.
  • For more information, see:
    Key facts
    - Active substance
    - Adeno-associated virus serotype rh10 containing the human GALC gene
    - Intended use
    - Treatment of Krabbe disease
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/21/2511
    - Date of designation
    - Sponsor
    Forge Biologics Europe S.L.

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

Retrieved on: 
Mardi, avril 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[...]

Retrieved on: 
Mardi, avril 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