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

Key Facts

PIPS Key Facts
Active Substance
Active Substance:
  • eladocagene exuparvovec
Invented Name
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
Decision Type
Decision Type
PM: decision on the application for modification of an agreed paediatric investigation plan.
Compliance Check
Compliance Check
Compliance Check Decision Date
Compliance Check Procedure Number

Decision Document

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