EMEA-001855-PIP01-15-M02 - paediatric investigation plan

Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (fitusiran)

Key facts

• Active Substance:
  Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (fitusiran)
• Therapeutic area:
  Haematology-Hemostaseology
• Decision number:
  P/0302/2020
• PIP number:
  EMEA-001855-PIP01-15-M02
• Pharmaceutical form(s):
  Subcutaneous use
• Condition(s) / indication(s):
  • Treatment of congenital haemophilia A
  • Treatment of congenital haemophilia B
• Route(s) of administration:
  Solution for injection
• Contact for public enquiries:

  Genzyme Europe B.V.

  Tel. [phone]; E-mail: [e-mail]

• Decision type:
  PM: decision on the application for modification of an agreed PIP
• Decision date:
  12/08/2020

Decision

P/0302/202: EMA decision of 12 August 2020 on the acceptance of a modification of an agreed paediatric investigation plan for synthetic doublestranded siRNA oligonucleotide...(EMEA-001855-PIP01-15-M02)

AdoptedReference Number: EMA/413460/2020