MHRA-102202-PIP01-25-M01 (update)

Key Facts

Active Substance
Active Substance:
  • ONASEMNOGENE ABEPARVOVEC
Invented Name
  • Zolgensma
  • Zolgensma
PIP Number MHRA-102202-PIP01-25-M01 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Solution for injection/infusion
Therapeutic area
Therapeutic area:
  • Neurology
Conditions / Indications
Conditions / Indications:
  • Treatment of spinal muscular atrophy.
Route(s) of administration
  • Intravenous use
  • INTRATHECAL 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
11/12/2025
Compliance Check Procedure Number
Compliance procedure number
MHRA-102202-PIP01-25-M01-C

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):ONASEMNOGENE ABEPARVOVEC.pdf
Published Date 16/01/2026