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
No
Compliance Check Decision Date
Compliance Check Procedure Number

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):eladocagene exuparvovec.pdf
Published Date 17/12/2021