EMEA-001244-PIP01-11-M02 - paediatric investigation plan

autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ATP-binding cassette, sub-family D (ALD), member 1 (ABCD1) from cDNA

Key facts

• Active substance:
  autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ATP-binding cassette, sub-family D (ALD), member 1 (ABCD1) from cDNA
• Therapeutic area:
  Neurology
• Decision number:
  P/0290/2018
• PIP number:
  EMEA-001244-PIP01-11-M02
• Pharmaceutical form(s):
  Dispersion for infusion
• Condition(s) / indication(s):
  Treatment of adrenoleukodystrophy
• Route(s) of administration:
  Intravenous use
• Decision type:
  PM: decision on the application for modification of an agreed PIP
• Decision date:
  12/09/2018
• Compliance check done:
  Yes
• Compliance procedure number:
  EMEA-C-001244-PIP01-11-M02
• Compliance opinion date:
  04/09/2020
• Compliance outcome:
  Positive

Decision

P/0290/2018: EMA decision of 12 September 2018 on the acceptance of a modification of an agreed paediatric investigation plan for autologous haematopoietic stem cells transduced with lentiviral vector... (EMEA-001244-PIP01-11-M02)

Adopted Reference Number: EMA/538560/2018