EMA

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

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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

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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

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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

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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

Orphan designation: cusatuzumab Treatment of acute myeloid leukaemia, 22/04/2020 Positive

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Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Clinical research, Health, B cell, Growth, Cell, Bone marrow, Infection, AML, Disease, Patient, Committee, Knowledge, Regulation, CD70, Cancer, EMA, IRIS, Business park, Acute myeloid leukemia, European, COMP, European Commission, Blood, Azacitidine, Safety, Haematopoietic system, Marketing, Acute leukemia, Medicine, Pharmaceutical industry

Orphan designation: cusatuzumab Treatment of acute myeloid leukaemia, 22/04/2020 Positive

Key Points: 


Orphan designation: cusatuzumab Treatment of acute myeloid leukaemia, 22/04/2020 Positive

Orphan designation: Upifitamab rilsodotin Treatment of ovarian cancer, 09/12/2022 Positive

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Thursday, April 18, 2024
Community, Diagnosis, Civil service commission, Ovarian cancer, Cell death, Prevalence, Cell division, Disease, Clinical, Patient, Committee, EMA, IRIS, 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 ovarian cancer in the European Union on 9 December 2022.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • Upifitamab rilsodotin is composed of an antibody that binds NaPi2b and a drug called AF-HPA which prevents cell division, resulting in cell death.
  • 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: (S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester Treatment of pulmonary arterial hypertension, 20/04/2017 Positive

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Thursday, April 18, 2024
Eurostat, Blood pressure, Diagnosis, Tadalafil, Blood, Serotonin, Patient, Committee, Knowledge, Regulation, Prostacyclin, EMA, IRIS, Artery, Life expectancy, Heart failure, Orthostatic hypertension, European, Therapy, COMP, Heart, Safety, European Commission, Marketing, Pulmonary hypertension, Medicine, Pharmaceutical industry

Orphan designation: (S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester Treatment of pulmonary arterial hypertension, 20/04/2017 Positive

Key Points: 


Orphan designation: (S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester Treatment of pulmonary arterial hypertension, 20/04/2017 Positive

Orphan designation: Diacerein Treatment of epidermolysis bullosa, 20/02/2014 Positive

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Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Gene, Anthraquinones, Skin cancer, Epidermolysis bullosa, Disease, Inflammation, Patient, Committee, Knowledge, Regulation, Keratin, EMA, IRIS, Joint, Protein, Life expectancy, Deformity, Blister, Interleukin-1 family, Mouth, European, Skin, Collagen, COMP, Infection, Safety, European Commission, Pain, Osteoarthritis, Marketing, Swelling, FGK, Medicine, Pharmaceutical industry

Orphan designation: Diacerein Treatment of epidermolysis bullosa, 20/02/2014 Positive

Key Points: 


Orphan designation: Diacerein Treatment of epidermolysis bullosa, 20/02/2014 Positive

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

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Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Death, Glycosaminoglycan, B cell, Enzyme, Mutation, Medicine, Cell, Mental disability, Disease, Patient, Committee, Knowledge, Scheie syndrome, Regulation, EMA, IRIS, Periodic breathing, Mucopolysaccharidosis, European, COMP, Enzyme replacement therapy, European Commission, Safety, DSM-IV codes, IQVIA, Marketing, Haematopoiesis, Liver, RDS, FGK, Hurler syndrome, MPS, Pharmaceutical industry

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Key Points: 


Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Cancer Medicines Forum: February 2024, Online, European Medicines Agency, Amsterdam, the Netherlands, 26 February 2024

Retrieved on: 
Thursday, April 18, 2024
Location, Cancer, EMA, CMF, Vaccine

Date

Key Points: 
  • Date
    - Monday, 26 February 2024
    Location
    - OnlineEuropean Medicines Agency, Amsterdam, the Netherlands
    Event summary
    The Cancer Medicines Forum (CMF) aims to explore how EMA can contribute towards addressing remaining uncertainties about the use of cancer medicines in clinical practice.
  • The forum brings together representatives of academic organisations from EMA’s Healthcare Professionals Working Party and the European medicines regulatory network.
  • The results of discussions will support the prioritisation of actions to fight cancer in EMA's Regulatory Science Strategy to 2025 and Academia Collaboration Matrix Action Plan.
  • The meetings of the forum are by invitation only.