IRIS

SPOR Status Update, Online, European Medicines Agency, Amsterdam, the Netherlands, Broadcast, from 10 July 2024, 10:00 (CEST) to 10 July 2024, 12:30 (CEST)

Retrieved on: 
Vendredi, avril 19, 2024
SMS, Location, RMS, OMS, IRIS, EMA, RDM

Date

Key Points: 
  • Date
    - Wednesday, 10 July 2024, 10:00 (CEST) - 12:30 (CEST)
    Location
    - OnlineEuropean Medicines Agency, Amsterdam, the NetherlandsLive Broadcast
    Event summary
    EMA is organising a public webinar on Substance, Product, Organisation, Referentials (SPOR) Regulatory Data Management (RDM) services status update.
  • SPOR RDM services aim to facilitate the reliable exchange of medicinal product information in a robust and consistent manner, enabling increased data quality and interoperability, efficiencies and more efficient regulatory action.
  • Through the integration of its services with other systems used for regulatory procedures, such as IRIS, SPOR actively contributes to their success and full benefits realisation.
  • SMS, XEVMPD/Art.57, OMS, and RMS) will be provided:
    - Achievements in Q2 2024.

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Retrieved on: 
Jeudi, avril 18, 2024
Eurostat, Diagnosis, Civil service commission, Absorption, Lower respiratory tract infection, Exercise, Cell, Infection, DSM-IV codes, Inflammation, Patient, Committee, Cystic fibrosis, Knowledge, Digestion, Regulation, Bacteria, Food, EMA, IRIS, Anti-inflammatory, Mucus, CB2, European, COMP, Pancreas, Sponsor, Ivacaftor, European Commission, Safety, CFTR, Marketing, Medicine, Pharmaceutical industry

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Key Points: 


Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Orphan designation: mRNA encoding the human CFTR gene Treatment of cystic fibrosis, 19/02/2024 Positive

Retrieved on: 
Jeudi, avril 18, 2024
Patient, Committee, Arcturus Therapeutics, Messenger, EMA, IRIS, European, Therapy, COMP, European Commission, CFTR, Marketing, Cystic fibrosis, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of cystic fibrosis in the European Union 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: (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: 
Jeudi, avril 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: 
Jeudi, avril 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: 
Jeudi, avril 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: 
Jeudi, avril 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: 
Jeudi, avril 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: 
Jeudi, avril 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: 
Jeudi, avril 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: