EU/3/23/2751 - orphan designation for treatment of glycogen storage disease type II (Pompe's disease)
autologous CD34+ cells transduced with a lentiviral vector containing the human GAA gene
OrphanHuman
Overview
This medicine was designated as an orphan medicine for the glycogen storage disease type II (Pompe's disease) in the European Union on 15 February 2023.
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:
Key facts
- Active substance
- autologous CD34+ cells transduced with a lentiviral vector containing the human GAA gene
- Intended use
- Treatment of glycogen storage disease type II (Pompe's disease)
- Orphan designation status
- Positive
- EU designation number
- EU/3/23/2751
- Date of designation
- Sponsor
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
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.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
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: