EMEA-002403-PIP01-18 - paediatric investigation plan

synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid (tofersen)

Key facts

• Active substance:
  synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid (tofersen)
• Therapeutic area:
  Neurology
• Decision number:
  P/0337/2018
• PIP number:
  EMEA-002403-PIP01-18
• Pharmaceutical form(s):
  Solution for injection/infusion
• Condition(s) / indication(s):
  Treatment of amyotrophic lateral sclerosis
• Route(s) of administration:
  Intrathecal use
• Decision type:
  W: decision granting a waiver in all age groups for all conditions or indications
• Decision date:
  08/10/2018
• Compliance check done:
  No

Decision

P/0337/2018: EMA decision of 8 October 2018 on the granting of a product specific waiver for synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 (SOD1) messenger ribonucleic acid (EMEA-002403-PIP01-18)

Adopted Reference Number: EMA/670855/2018