MHRA-100374-PIP01-21

Key Facts

Active Substance
Active Substance:
  • Adeno-associated virus serotype 1 vector containing the human progranulin GRN gene
Invented Name
Not yet available
PIP Number MHRA-100374-PIP01-21
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Solution for injection
Therapeutic area
Therapeutic area:
  • Neurology
Conditions / Indications
Conditions / Indications:
  • Treatment of frontotemporal dementia
Route(s) of administration
Route(s) of administration:
  • Intracisternal use
PIP applicant
Decision Type
Decision Type
W: decision granting a waiver in all age groups for the listed condition(s).
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):Adeno-associated virus serotype 1 vector containing the human progranulin GRN gene.pdf
Published Date 27/09/2022