EMEA-002779-PIP01-20 - paediatric investigation plan

Adeno-associated virus, serotype 9 (AAV9)-based non-replicating, self-complementary recombinant vector containing an expression cassette for the human ASPA transgene (scAAV9-CB6-hASPAopt)

Key facts

• Active Substance:
  Adeno-associated virus, serotype 9 (AAV9)-based non-replicating, self-complementary recombinant vector containing an expression cassette for the human ASPA transgene (scAAV9-CB6-hASPAopt)
• Therapeutic area:
  Neurology
• Decision number:
  P/0128/2021
• PIP number:
  EMEA-002779-PIP01-20
• Pharmaceutical form(s):
  Solution for injection
• Condition(s) / indication(s):
  Treatment of Canavan disease
• Route(s) of administration:
  Intravenous use
• Contact for public enquiries:

  Aspa Therapeutics, Inc.

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

• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Decision date:
  14/04/2021

Decision

P/0128/2021 : EMA decision on the agreement of a PIP and on the granting of a deferral for adenoassociated virus, serotype 9 (AAV9)-based non-replicating, self-complementary recombinant vector containing an expression cassette for the human ASPA transgene

Reference Number: EMA/151831/2021