EMEA-001974-PIP01-16-M02 - paediatric investigation plan

autologous CD34+ hematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for the human adenosine deaminase gene

Key facts

• Active substance:
  autologous CD34+ hematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for the human adenosine deaminase gene
• Therapeutic area:
  Immunology-Rheumatology-Transplantation
• Decision number:
  P/0400/2018
• PIP number:
  EMEA-001974-PIP01-16-M02
• Pharmaceutical form(s):
  Dispersion for infusion
• Condition(s) / indication(s):
  Treatment of severe combined immunodeficiency due to adenosine deaminase deficiency
• Route(s) of administration:
  Intravenous use
• Decision type:
  PM: decision on the application for modification of an agreed PIP
• Decision date:
  26/11/2018
• Compliance check done:
  No

Decision

P/0400/2018: EMA decision of 26 November 2018 on the acceptance of a modification of an agreed paediatric investigation plan for autologous CD34+ hematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for the human adenosine deam

Adopted Reference Number: EMA/762335/2018