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

Overview

Metadata

Provider: 

European Medicines Agency

URL: 

https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2894

Retrieved on: 

Thursday, April 18, 2024

Information

Tags: 

Patient, Committee, Arcturus Therapeutics, Messenger, EMA, IRIS, European, Therapy, COMP, European Commission, CFTR, Marketing, Cystic fibrosis, Medicine, Pharmaceutical industry

Geotags: 

EUROPEAN UNION, RARE, EU

Summary

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:

Copyright: 

2024 European Medicines Agency. All Rights Reserved.