EMEA-003639-PIP01-24 - paediatric investigation plan

adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene (RP-A501)

Key facts

• Active substance:
  adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene (RP-A501)
• Therapeutic area:
  Congenital, familial and genetic disorders
• Decision number:
  P/0004/2025
• PIP number:
  EMEA-003639-PIP01-24
• Pharmaceutical form(s):
  Solution for infusion
• Condition(s) / indication(s):
  Treatment of Danon Disease
• 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:
  27/01/2025
• Compliance check done:
  No

Decision

P/0004/2025 : EMA decision of 27 January 2025 on the agreement of a PIP and on the granting of a deferral and waiver for adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene (RP-A501) (EMEA-003639-PIP01-24)

Adopted Reference Number: EMA/25307/2025