Orphan designation: Adeno-associated virus serotype rh10 containing the human GALC gene Treatment of Krabbe disease, 15/10/2021 Positive

Overview

Metadata

Provider: 

European Medicines Agency

URL: 

https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2511

Retrieved on: 

Tuesday, April 9, 2024

Information

Tags: 

Diamond, Community, Diagnosis, Civil service commission, Prevalence, Disease, Patient, Committee, EMA, IRIS, Krabbe disease, GALC, Agency, European, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Geotags: 

EUROPEAN UNION, RARE, NETHERLANDS, EU

Summary

Key Points: 

• Overview
  This medicine was designated as an orphan medicine for the treatment of Krabbe disease in the European Union on 15 October 2021.
• All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
• The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
• For more information, see:
  Key facts
  - Active substance
  - Adeno-associated virus serotype rh10 containing the human GALC gene
  - Intended use
  - Treatment of Krabbe disease
  - Orphan designation status
  - Positive
  - EU designation number
  - EU/3/21/2511
  - Date of designation
  - Sponsor
  Forge Biologics Europe S.L.

Copyright: 

2024 European Medicines Agency. All Rights Reserved.