Eplontersen (1) – Wainzua®

• Clinical trials
  • The NEURO-TTRansform study, on which the marketing authorisation is based, is a randomised, open-label, Phase III study with a one-way crossover design.

Adults with hereditary transthyretin amyloidosis with stage 1 or 2 polyneuropathy

• For adults with hereditary transthyretin amyloidosis with stage 1 or 2 polyneuropathy, the additional benefit is not proven.
• For adults with hereditary transthyretin amyloidosis with stage 1 or 2 polyneuropathy, the additional benefit of eplontersen compared with the appropriate comparator therapy is not proven.
• No relevant study was identified for the assessment of the additional benefit of eplontersen compared with the appropriate comparator therapy.
• The study compares Eplontersen with Inotersen. Consequently, no comparative data are available against the appropriate comparator therapy, Vutrisiran.
• Even though Inotersen, like Vutrisiran, is a TTR silencer, a comparison of eplontersen with inotersen – contrary to the pharmaceutical manufacturer’s assessment – does not allow any conclusions to be drawn regarding the classification of the additional benefit of eplontersen compared with the appropriate comparator therapy, vutrisiran.

Courtesy translation only, please refer to the German original.