MHRA-100129-PIP01-21-M02 (update)

Key Facts

Active Substance
Active Substance:
  • Cipaglucosidase alfa
Invented Name
  • Pombiliti
  • Pombilti
  • Pombiliti
PIP Number MHRA-100129-PIP01-21-M02 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Powder for concentrate for solution for infusion
Therapeutic area
Therapeutic area:
  • Endocrinology-Gynaecology-Fertility-metabolism
Conditions / Indications
Conditions / Indications:
  • Treatment of glycogen storage disease Type II (Pompe's disease)
Route(s) of administration
Route(s) of administration:
  • Intravenous use
PIP applicant
  • Amicus Therapeutics UK Limited
  • Country United Kingdom
  • Tel +16096623809
  • Email IR@amicusrx.com
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):CIPAGLUCOSIDASE ALFA.pdf
Published Date 08/04/2025