EMEA-003420-PIP01-23 - paediatric investigation plan

Synthetic double-stranded siRNA oligonucleotide directed against apolipoprotein C-III mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues

Key facts

• Active Substance:
  Synthetic double-stranded siRNA oligonucleotide directed against apolipoprotein C-III mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
• Therapeutic area:
  Congenital, familial and genetic disorders
• Decision number:
  P/0071/2024
• PIP number:
  EMEA-003420-PIP01-23
• Pharmaceutical form(s):
  Concentrate for solution for injection
• Condition(s) / indication(s):
  Treatment of familial chylomicronaemia syndrome (type I hyperlipoproteinaemia
• Route(s) of administration:
  Subcutaneous use
• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Decision date:
  08/03/2024

Decision

P/0071/2024 : EMA decision of 8 March 2024 on the agreement of a paediatric investigation plan and on the granting of a deferral and on the granting of a waiver for synthetic double-stranded siRNA oligonucleotide... (EMEA-003420-PIP01-23)

Reference Number: EMA/28239/2024