MHRA-101149-PIP01-23-M01 (update)

Key Facts

Active Substance
Active Substance:
  • elosulfase alfa (recombinant human N-acetylgalactosamine-6-sulfatase)
Invented Name
Vimizim 1mg/ml concentrate for solution for infusion
PIP Number MHRA-101149-PIP01-23-M01 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Concentrate for solution for infusion (Sterile concentrate)
Therapeutic area
Therapeutic area:
  • Endocrinology-Gynaecology-Fertility-metabolism
Conditions / Indications
Conditions / Indications:
  • Treatment of mucopolysaccharidosis, type IVA (Morquio-A syndrome)
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
Yes
Compliance Check Decision Date
Compliance opinion date
21/09/2023
Compliance Check Procedure Number
Compliance procedure number
MHRA-101149-PIP01-23-M01-C

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):elosulfase alfa (recombinant human N-acetylgalactosamine-6-sulfatase).pdf
Published Date 19/12/2023