MHRA-100330-PIP01-21 -M02 (update)

Key Facts

Active Substance
Active Substance:
  • Azamidugene autotemcel (OTL-203)
Invented Name
Not available at present
PIP Number MHRA-100330-PIP01-21 -M02 (update)
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
PM: decision on the application for modification of an agreed paediatric investigation plan.
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):Azamidugene autotemcel (OTL-203).pdf
Published Date 08/04/2025