MHRA-100097-PIP01-21-M01 (update)

Key Facts

Active Substance
Active Substance:
  • eladocagene exuparvovec
Invented Name
Upstaza
PIP Number MHRA-100097-PIP01-21-M01 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Solution for infusion
Therapeutic area
Therapeutic area:
  • Neurology
Conditions / Indications
Conditions / Indications:
  • Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
Route(s) of administration
Route(s) of administration:
  • Intracerebral use
PIP applicant
  • PTC Therapeutics International Limited
  • Country Ireland
  • Tel +35319068700
  • Email medinfo@ptcbio.com
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
19/07/2022
Compliance Check Procedure Number
Compliance procedure number
MHRA-100097-PIP01-21-M01-C

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):eladocagene exuparvovec.pdf
Published Date 14/04/2026