EMA/PE/0000232896 - paediatric investigation plan

antisense oligonucleotide against patatin like phospholipase domain containing protein 3 gene (PNPLA3), conjugated to N-acetylgalactosamine (AZD2693), sodium salt

Key facts

• Active substance:
  antisense oligonucleotide against patatin like phospholipase domain containing protein 3 gene (PNPLA3), conjugated to N-acetylgalactosamine (AZD2693), sodium salt
• Therapeutic area:
  Hepatobiliary disorders
• Decision number:
  EMA/PE/0000232896
• PIP number:
  EMA/PE/0000232896
• Pharmaceutical form(s):
  Solution for injection
• Condition(s) / indication(s):
  Treatment of metabolic dysfunction-associated steatohepatitis
• Route(s) of administration:
  Subcutaneous use
• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Decision date:
  21/02/2025
• Compliance check done:
  No

Decision

EMA/PE/0000232896 : EMA decision of 21 February 2025 on the agreement of a paediatric investigation plan and on the granting of a deferral and on the granting of a waiver for antisense oligonucleotide against patatin like phospholipase....sodium salt

Adopted Reference Number: EMADOC-1700519818-1911730