MHRA-100891-PIP01-23

Key Facts

Active Substance
Active Substance:
  • MODIFIED MESSENGER RIBONUCLEIC ACID ENCODING HUMAN PROPIONYL-COENZYME A CARBOXYLASE ALPHA AND BETA SUBUNITS ENCAPSULATED INTO LIPID NANOPARTICLES
Invented Name
Not yet available
PIP Number MHRA-100891-PIP01-23
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Dispersion for injection
Therapeutic area
Therapeutic area:
  • Endocrinology-Gynaecology-Fertility-metabolism
Conditions / Indications
Conditions / Indications:
  • Treatment of propionic acidaemia
Route(s) of administration
Route(s) of administration:
  • Intravenous 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):MODIFIED MESSENGER RIBONUCLEIC ACID ENCODING HUMAN PROPIONYL-COENZYME A CARBOXYLASE ALPHA AND BETA SUBUNITS ENCAPSULATED INTO LIPID NANOPARTICLES.pdf
Published Date 28/10/2024