EMEA-003001-PIP01-21-M01 - paediatric investigation plan

autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203)

Key facts

• Active substance:
  autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203)
• Therapeutic area:
  Other
• Decision number:
  P/0167/2023
• PIP number:
  EMEA-003001-PIP01-21-M01
• Pharmaceutical form(s):
  Dispersion for infusion
• Condition(s) / indication(s):
  Treatment of mucopolysaccharidosis type I, Hurler syndrome
• Route(s) of administration:
  Intravenous use
• Decision type:
  PM: decision on the application for modification of an agreed PIP
• Decision date:
  15/05/2023
• Compliance check done:
  No

Decision

P/0167/2023 : EMA decision of 15 May 2023 on the acceptance of a modification of an agreed paediatric investigation plan for autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified ... (EMEA-003001-PIP01-21-M01)

Reference Number: EMA/160450/2023