MHRA-101824-PIP01-25

Key Facts

Active Substance
Active Substance:
  • AAV8-RK-AIPL1
Invented Name
Not available at present
PIP Number MHRA-101824-PIP01-25
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Suspension for injection
Therapeutic area
Therapeutic area:
  • Ophthamology
Conditions / Indications
Conditions / Indications:
  • Treatment of Leber congenital amaurosis
Route(s) of administration
Route(s) of administration:
  • INTRAOCULAR USE
PIP applicant
Decision Type
Decision Type
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s)
Compliance Check
Compliance Check
Yes
Compliance Check Decision Date
Compliance opinion date
03/12/2025
Compliance Check Procedure Number
Compliance procedure number
MHRA-101824-PIP01-25-C

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):AAV8-RK-AIPL1.pdf
Published Date 18/12/2025