EMEA-000973-PIP01-10-M04 - paediatric investigation plan

recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa)

Key facts

• Invented name:
  Vimizim
• Active substance:
  recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa)
• Therapeutic area:
  Endocrinology-Gynaecology-Fertility-Metabolism
• Decision number:
  P/0170/2023
• PIP number:
  EMEA-000973-PIP01-10-M04
• Pharmaceutical form(s):
  Concentrate for solution for infusion
• Condition(s) / indication(s):
  Treatment of mucopolysaccharidosis, type IVA (Morquio-A syndrome)
• Route(s) of administration:
  Intravenous use
• Decision type:
  PM: decision on the application for modification of an agreed PIP
• Decision date:
  15/05/2023
• Compliance check done:
  Yes
• Compliance procedure number:
  EMEA-C-000973-PIP01-10-M04
• Compliance opinion date:
  23/06/2023
• Compliance outcome:
  Positive

Decision

P/0170/2023 : EMA decision of 15 May 2023 on the acceptance of a modification of an agreed paediatric investigation plan for elosulfase alfa (Vimizim), (EMEA-000973-PIP01-10-M04)

Adopted Reference Number: EMA/170999/2023