EMEA-002021-PIP01-16-M01 - paediatric investigation plan

rAAV8 viral vector encoding the human UGT1A1 transgene (rAAV8-hUGT1A1)

Key facts

• Active substance:
  rAAV8 viral vector encoding the human UGT1A1 transgene (rAAV8-hUGT1A1)
• Therapeutic area:
  Congenital, familial and genetic disorders
• Decision number:
  P/0228/2024
• PIP number:
  EMEA-002021-PIP01-16-M01
• Pharmaceutical form(s):
  Concentrate for solution for infusion
• Condition(s) / indication(s):
  Treatment of Crigler-Najjar syndrome
• Route(s) of administration:
  Intravenous use
• Decision type:
  PM: decision on the application for modification of an agreed PIP
• Decision date:
  19/07/2024
• Compliance check done:
  No

Decision

P/0228/2024: EMA decision of 19 July 2024 on the acceptance of a modification of an agreed paediatric investigation plan for rAAV8 viral vector encoding the human UGT1A1 transgene (rAAV8-hUGT1A1) (EMEA-002021-PIP01-16-M01)

Adopted Reference Number: EMA/291508/2024