MHRA-102230-PIP01-25-M01 (update)

Key Facts

Active Substance
Active Substance:
  • Rebisufligene etisparvovec
Invented Name
Not available at present
PIP Number MHRA-102230-PIP01-25-M01 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Solution for infusion
Therapeutic area
Therapeutic area:
  • Neurology
Conditions / Indications
Conditions / Indications:
  • Treatment of mucopolysaccharidosis IIIA
Route(s) of administration
Route(s) of administration:
  • Parenteral 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
11/03/2026
Compliance Check Procedure Number
Compliance procedure number
MHRA-102230-PIP01-25-M01-C1

Decision Document

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