MHRA-100482-PIP01-22-M01 (update)

Key Facts

Active Substance
Active Substance:
  • acetyl-L-leucine ((s)-(acetylamino)-4-methylpentanoic acid)
Invented Name
Not available at present
PIP Number MHRA-100482-PIP01-22-M01 (update)
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Age-appropriate oral liquid dosage form.
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
No
Compliance Check Decision Date
Compliance Check Procedure Number

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):acetyl-L-leucine ((s)-(acetylamino)-4-methylpentanoic acid).pdf
Published Date 19/12/2023