MHRA-102165-PIP01-25

Key Facts

Active Substance
  • adeno-associated viral vector serotype Anc80 containing the 5' portion of human OTOF gene
  • adeno-associated viral vector serotype Anc80 containing the 3' portion of human OTOF gene
Invented Name
Not yet available
PIP Number MHRA-102165-PIP01-25
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Suspension for injection
Therapeutic area
Therapeutic area:
  • Oto-Rhino-Laryngology
Conditions / Indications
Conditions / Indications:
  • Treatment of otoferlin gene-mediated hearing loss
Route(s) of administration
Route(s) of administration:
  • Auricular 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 Check Procedure Number
Compliance procedure number
MHRA-102165-PIP01-25-C1

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):adeno-associated viral vector serotype Anc80 containing the 5' portion of human OTOF geneadeno-associated viral vector serotype Anc80 containing the 3' portion of human OTOF gene.pdf
Published Date 08/01/2026