EMEA-003524-PIP01-23 - paediatric investigation plan

adeno‐associated viral vector serotype 8 containing the 3' human otoferlin coding sequence / adeno‐associated viral vector serotype 8 containing the 5' human otoferlin coding sequence

Key facts

• Active substance:
  adeno‐associated viral vector serotype 8 containing the 3' human otoferlin coding sequence / adeno‐associated viral vector serotype 8 containing the 5' human otoferlin coding sequence
• Therapeutic area:
  Congenital, familial and genetic disorders
• Decision number:
  P/0340/2024
• PIP number:
  EMEA-003524-PIP01-23
• Pharmaceutical form(s):
  Solution for injection
• Condition(s) / indication(s):
  Treatment of otoferlin gene-mediated hearing loss
• Route(s) of administration:
  Auricular use
• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Decision date:
  11/09/2024
• Compliance check done:
  No

Decision

P/0340/2024 : EMA decision of 11 September 2024 on the agreement of a paediatric investigation plan and on the granting of a deferral and on the granting of a waiver for adeno‐associated viral vector serotype 8 containing the 3'....(EMEA-003524-PIP01-23)

Adopted Reference Number: EMA/414602/2024