EMEA-002734-PIP01-19-M01 - paediatric investigation plan

adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (DTX401)

Key facts

• Active substance:
  adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (DTX401)
• Therapeutic area:
  Congenital, familial and genetic disorders
• Decision number:
  P/0044/2024
• PIP number:
  EMEA-002734-PIP01-19-M01
• Pharmaceutical form(s):
  Concentrate and diluent for solution for infusion
• Condition(s) / indication(s):
  Treatment of glycogen storage disease type Ia
• Route(s) of administration:
  Intravenous use
• Decision type:
  PM: decision on the application for modification of an agreed PIP
• Decision date:
  15/02/2024
• Compliance check done:
  No

Decision

P/0044/2024 : EMA decision of 15 February 2024 on the acceptance of a modification of an agreed paediatric investigation plan for pariglasgene brecaparvovec (DTX401) (EMEA-002734-PIP01-19-M01)

Adopted Reference Number: EMA/29056/2024