MHRA-100129-PIP01-21-M01 (update)

Key Facts

Active Substance
Active Substance:
  • Cipaglucosidase alfa
Invented Name
Pombiliti
PIP Number MHRA-100129-PIP01-21-M01 (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 0017326668271
  • Email dmoore@amicusrx.com
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 Check Procedure Number
Compliance procedure number
MHRA-100129-PIP01-21-M01-C1

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):Cipaglucosidase alfa.pdf
Published Date 29/07/2022