European Commission

Orphan designation: Treatment of small cell lung cancer, 19/02/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
Carcinoma, Patient, Committee, EMA, IRIS, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of small lung cell cancer on 19 February 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • 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: Raludotatug deruxtecan Treatment of ovarian cancer, 19/02/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
Ovarian cancer, Patient, Committee, EMA, IRIS, 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 on 19 February 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • 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: Andecaliximab Treatment of fibrodysplasia ossificans progressiva, 19/02/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
European Commission, Patient, Committee, Marketing, COMP, Medicine, IRIS, EMA, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of fibrodysplasia ossificans progressiva on 19 February 2024.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • For information on ongoing clinical trials in the EU, see:
    Key facts
    - Active substance
    - Andecaliximab
    - Intended use
    - Treatment of fibrodysplasia ossificans progressiva
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/24/2901
    - Date of designation
    - Sponsor
    Regulatory Pharma Net S.r.l.
  • 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: Repagermanium Treatment of focal segmental glomerulosclerosis, 19/02/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
Patient, Committee, EMA, IRIS, Focal segmental glomerulosclerosis, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of focal segmental glomerulosclerosis on 19 February 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • 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 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