EMEA-001502-PIP01-13 - paediatric investigation plan

adeno-associated viral vector serotype 9 containing human sulfamidase gene

Key facts

• Active Substance:
  adeno-associated viral vector serotype 9 containing human sulfamidase gene
• Therapeutic area:
  Endocrinology-Gynaecology-Fertility-Metabolism
• Decision number:
  P/0149/2014
• PIP number:
  EMEA-001502-PIP01-13
• Pharmaceutical form(s):
  Solution for injection/infusion
• Condition(s) / indication(s):
  Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
• Route(s) of administration:
  Intracerebroventricular use
• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Decision date:
  13/06/2014

Decision

P/0149/2014: EMA decision of 13 June 2014 on the agreement of a paediatric investigation plan and on the granting of a deferral and on the granting of a waiver for adeno-associated viral vector serotype 9 containing huma...

AdoptedReference Number: EMA/330057/2014