EMEA-003298-PIP01-22 - paediatric investigation plan

Messenger RNA encoding Cas9 and single guide RNA targeting the human TTR gene

Key facts

• Active substance:
  Messenger RNA encoding Cas9 and single guide RNA targeting the human TTR gene
• Therapeutic area:
  • Cardiovascular diseases
  • Neurology
• Decision number:
  P/0532/2022
• PIP number:
  EMEA-003298-PIP01-22
• Pharmaceutical form(s):
  Suspension for infusion
• Condition(s) / indication(s):
  Treatment of transthyretin amyloidosis (ATTR)
• Route(s) of administration:
  Intravenous use
• Decision type:
  W: decision granting a waiver in all age groups for all conditions or indications
• Decision date:
  30/12/2022
• Compliance check done:
  No

Decision

P/0532/2022 : EMA decision of 30 December 2022 on the granting of a product specific waiver for messenger RNA encoding Cas9 and single guide RNA targeting the human TTR gene (EMEA-003298-PIP01-22)

Adopted Reference Number: EMA/929798/2022