MHRA-100274-PIP01-21-M02 (update)

Key Facts

Active Substance
Active Substance:
  • ELIGLUSTAT
Invented Name
CERDELGA
PIP Number MHRA-100274-PIP01-21-M02 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Capsule, hard
Therapeutic area
Therapeutic area:
  • Other: Lysosomal Storage Disorder
Conditions / Indications
  • Treatment of Gaucher Disease Type 2
  • Treatment of Gaucher Disease Type 1 and Type 3
Route(s) of administration
Route(s) of administration:
  • Oral 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
02/02/2024
Compliance Check Procedure Number
Compliance procedure number
MHRA-100274-PIP01-21-M02-C1

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):ELIGLUSTAT.pdf
Published Date 14/04/2026