MHRA-100342-PIP01-21-M02 (update)

Key Facts

Active Substance
Active Substance:
  • arimoclomol citrate
Invented Name
MIPLYFFA
PIP Number MHRA-100342-PIP01-21-M02 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Capsule, hard
Therapeutic area
Therapeutic area:
  • Neurology
Conditions / Indications
Conditions / Indications:
  • Treatment of Niemann-Pick disease type C
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
06/01/2026
Compliance Check Procedure Number
Compliance procedure number
MHRA-100342-PIP01-21-M02-C

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):arimoclomol citrate.pdf
Published Date 01/05/2026