MHRA-101573-PIP01-24

Key Facts

Active Substance
Active Substance:
  • Trofinetide
Invented Name
  • Not yet available in the United Kingdom (Daybu proposed in the EEA and currently being assessed)
  • Daybue
PIP Number MHRA-101573-PIP01-24
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Oral solution
Therapeutic area
Therapeutic area:
  • Neurology
Conditions / Indications
Conditions / Indications:
  • Treatment of Rett syndrome
Route(s) of administration
  • Oral use
  • Gastroenteral use
PIP applicant
Decision Type
Decision Type
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s)
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):Trofinetide.pdf
Published Date 06/01/2025