MHRA-100267-PIP01-21-M01 (update)

Key Facts

Active Substance
Active Substance:
  • EXAGAMGLOGENE AUTOTEMCEL
Invented Name
Casgevy
PIP Number MHRA-100267-PIP01-21-M01 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Dispersion for infusion
Therapeutic area
Therapeutic area:
  • Haematology-Hemostaseology
Conditions / Indications
Conditions / Indications:
  • Treatment of beta-thalassemia intermedia and major
Route(s) of administration
Route(s) of administration:
  • Intravenous use
PIP applicant
Decision Type
Decision Type
PM: decision on the application for modification of an agreed paediatric investigation plan.
Compliance Check
Compliance Check
Yes
Compliance Check Decision Date
Compliance opinion date
24/03/2026
Compliance Check Procedure Number
Compliance procedure number
MHRA-100267-PIP01-21-M01-C1

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):EXAGAMGLOGENE AUTOTEMCEL.pdf
Published Date 10/04/2026