EMEA-002571-PIP01-19 - paediatric investigation plan

adeno-associated viral vector serotype 8 containing the human MTM1 gene (resamirigene bilparvovec)

Key facts

• Active substance:
  adeno-associated viral vector serotype 8 containing the human MTM1 gene (resamirigene bilparvovec)
• Therapeutic area:
  Other
• Decision number:
  P/0309/2020
• PIP number:
  EMEA-002571-PIP01-19
• Pharmaceutical form(s):
  Solution for infusion
• Condition(s) / indication(s):
  Treatment of X linked myotubular myopathy
• Route(s) of administration:
  Intravenous use
• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Decision date:
  14/08/2020
• Compliance check done:
  No

Decision

P/0309/2020 : EMA decision of 14 August 2020 on the agreement of a paediatric investigation plan and on the granting of a deferral for resamirigene bilparvovec (EMEA-002571-PIP01-19)

Adopted Reference Number: EMA/413764/2020