European Commission

Orphan designation: (4R)-3-(4-fluoro-2-hydroxyphenyl)-4-methyl-4,5-dihydro-1H-pyrazole-1-carboximidamide hydrochloride Treatment of systemic sclerosis, 12/01/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
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 systemic sclerosis on 12 January 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: Human coagulation factor X Treatment of acquired factor X deficiency, 12/01/2024 Positive

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

EU/3/23/2886 - orphan designation for treatment of acquired factor X deficiency

Key Points: 
  • EU/3/23/2886 - orphan designation for treatment of acquired factor X deficiency
    Human coagulation factor X
    OrphanHuman
    BPL Bioproducts Laboratory GmbH
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Orphan designation: golcadomide hydrochloride Treatment of diffuse large B-cell lymphoma, 12/01/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
B-cell lymphoma, 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 diffuse large B-cell lymphoma on 12 January 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: Methyl-(1-{[6-{[(1S)-1-cyclopropylethyl]amino}-2-(pyrazolo[5,1-b][1,3]thiazol-7-yl)-pyrimidin-4-yl]carbonyl}piperidin-4-yl)carbamate mono(4-methylbenzenesulfonate) Treatment of eosinophilic granulomatosis with polyangiitis, 12/01/[...]

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

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of eosinophilic granulomatosis with polyangiitis on 12 January 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: Ersodetug Treatment of insulinoma, 12/01/2024 Positive

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

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of insulinoma on 12 January 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: Thiophene methylimidazole pentahydrogen Diagnosis of glioma, 12/01/2024 Positive

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

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the diagnosis of glioma on 12 January 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: mitapivat sulfate Treatment of thalassaemia intermedia and major, 12/01/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
Patient, Committee, Thalassemia, 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 thalassaemia alpha intermedia and major on 12 January 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: motixafortide Treatment of patients undergoing haematopoietic stem cell transplantation, 12/01/2024 Positive

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

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of patients undergoing haematopoietic stem cell transplantation on 12 January 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: Tarlatamab Treatment of small cell lung cancer, 12/01/2024 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 cell lung cancer on 12 January 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
    - Tarlatamab
    - Intended use
    - Treatment of small cell lung cancer
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2876
    - Date of designation
    - Sponsor
    Minervum 7061
    4817 ZK Breda
    The Netherlands
    Tel.
  • 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: cutamesine alpha-thalassaemia X-linked intellectual disability syndrome (due to mutations in the ATRX gene), 12/01/2024 Positive

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

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of alpha-thalassaemia X-linked intellectual disability syndrome (due to mutations in the ATRX gene) on 12 January 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: