EU/3/24/3020 - orphan designation for inherited retinal dystrophy due to defects in the RPE65 gene

Adeno-associated virus serotype 9 containing the human RPE65 gene

Overview

This medicine was designated as an orphan medicine for the treatment of inherited retinal dystrophy due to defects in the RPE65 gene in the European Union on 16 January 2025.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

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.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see: 

• EU Clinical Trials Register

• ClinicalTrials.gov 

Key facts

• Active substance:
  Adeno-associated virus serotype 9 containing the human RPE65 gene
• Intended use:
  inherited retinal dystrophy due to defects in the RPE65 gene
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/24/3020
• Date of designation:
  16/01/2025
• Sponsor:

  Granzer Regulatory Consulting & Services GmbH;