MHRA-101283-PIP01-23-M01 (update)

Key Facts

Active Substance
Active Substance:
  • Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Invented Name
Not yet available
PIP Number MHRA-101283-PIP01-23-M01 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Solution for injection
Therapeutic area
Therapeutic area:
  • Haematology-Hemostaseology
Conditions / Indications
  • Treatment of congenital haemophilia A
  • Treatment of congenital haemophilia B.
Route(s) of administration
Route(s) of administration:
  • Subcutaneous use
PIP applicant
Decision Type
Decision Type
PM: decision on the application for modification of an agreed paediatric investigation plan.
Compliance Check
Compliance Check
No
Compliance Check Decision Date
Compliance Check Procedure Number

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues.pdf
Published Date 24/06/2024