MHRA-100885-PIP01-23-M01 (update)

Key Facts

Active Substance
Active Substance:
  • IPTACOPAN HYDROCHLORIDE MONOHYDRATE
Invented Name
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
  • Fabhalta
PIP Number MHRA-100885-PIP01-23-M01 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Capsule, hard Age appropriate formulation
Therapeutic area
  • Haematology-Hemostaseology
  • Other: Complement inhibitor
Conditions / Indications
Conditions / Indications:
  • Treatment of paroxysmal nocturnal haemoglobinuria (PNH)
Route(s) of administration
Route(s) of administration:
  • Oral 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):IPTACOPAN HYDROCHLORIDE MONOHYDRATE.pdf
Published Date 18/12/2025