MHRA-101913-PIP01-25

Key Facts

Active Substance
Active Substance:
  • antisense oligonucleotide against patatin like phospholipase domain containing protein 3 gene (PNPLA3), conjugated to N-acetylgalactosamine (AZD2693), sodium salt
Invented Name
Not available at present
PIP Number MHRA-101913-PIP01-25
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Solution for injection
Therapeutic area
Therapeutic area:
  • Gastroenterology-Hepatology
Conditions / Indications
Conditions / Indications:
  • Treatment of metabolic dysfunction-associated steatohepatitis (MASH)
Route(s) of administration
Route(s) of administration:
  • Subcutaneous use
PIP applicant
Decision Type
Decision Type
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s)
Compliance Check
Compliance Check
No
Compliance Check Decision Date
Compliance Check Procedure Number

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):antisense oligonucleotide against patatin like phospholipase domain containing protein 3 gene (PNPLA3), conjugated to N-acetylgalactosamine (AZD2693), sodium salt.pdf
Published Date 18/12/2025