Orphan designation: Autologous CD34+ cells transduced with a lentiviral RNA vector that results in integrated cDNA encoding for functional cystinosin Treatment of cystinosis, 19/02/2021 Positive

All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

Metadata
Provider: 
European Medicines AgencyEuropean Medicines Agency
URL: 
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2407https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2407
Retrieved on: 
Tuesday, April 9, 2024
Information
Tags: 
Community, Diagnosis, Civil service commission, Prevalence, Cell, Bone marrow, Disease, Novartis, Patient, Committee, RNA, Autotransplantation, EMA, IRIS, Cystinosis, Agency, European, COMP, Blood, European Commission, Marketing, Medicine, Pharmaceutical industry
Geotags: 
EUROPEAN UNION, RARE, EU, BULGARIA
Summary
Key Points: 
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The medicine, also known as AVR-RD-O4, is a gene therapy, produced from immature blood (stem) cells collected from the patient.
  • After infusion into the patient, these cells permanently settle in the bone marrow, where they multiply to produce mature blood cells.
  • 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:
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