MHRA-100330-PIP01-21

Key Facts

Active Substance
Active Substance:
  • Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LVV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203)
Invented Name
Not yet available
PIP Number MHRA-100330-PIP01-21
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Dispersion for infusion
Therapeutic area
Therapeutic area:
  • Other: Metabolic disorders
Conditions / Indications
Conditions / Indications:
  • Treatment of Mucopolysaccharidosis type I, Hurler syndrome (MPS-IH)
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):Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LVV, encoding for the human ?-L-iduronidase (IDUA) gene (OTL-203).pdf
Published Date 29/07/2022