EMEA-003465-PIP01-23 - paediatric investigation plan

messenger RNA encoding Cas9, single guide RNA targeting the human KLKB1 gene

Key facts

• Active substance:
  messenger RNA encoding Cas9, single guide RNA targeting the human KLKB1 gene
• Therapeutic area:
  Congenital, familial and genetic disorders
• Decision number:
  P/0142/2024
• PIP number:
  EMEA-003465-PIP01-23
• Pharmaceutical form(s):
  Dispersion for infusion
• Condition(s) / indication(s):
  Treatment of hereditary angioedema
• 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:
  06/05/2024
• Compliance check done:
  No

Decision

P/0142/2024: EMA decision of 6 May 2024 on the agreement of a PIP and on the granting of a deferral and on the granting of a waiver for messenger RNA encoding Cas9, single guide RNA targeting the human KLKB1 gene (NTLA-2002) (EMEA-003465-PIP01-23)

Adopted Reference Number: EMA/153131/2024