EMEA-002206-PIP02-19 - paediatric investigation plan

Rebisufligene etisparvovec

Key facts

• Active Substance:
  Rebisufligene etisparvovec
• Therapeutic area:
  Endocrinology-Gynaecology-Fertility-Metabolism
• Decision number:
  P/0366/2020
• PIP number:
  EMEA-002206-PIP02-19
• Pharmaceutical form(s):
  Suspension for injection
• Condition(s) / indication(s):
  Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
• Route(s) of administration:
  Intravenous use
• Contact for public enquiries:

  Ultragenyx Germany GmbH

  E-mail: [e-mail]; Tel. : [phone]

• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Decision date:
  09/09/2020

Decision

P/0366/2020 : EMA decision of 9 September 2020 on the agreement of a paediatric investigation plan and on the granting of a deferral for rebisufligene etisparvovec (EMEA-002206-PIP02-19)

AdoptedReference Number: EMA/458002/2020