Diflunisal (1) – Attrogy®

• Clinical trials
  • Study H-23750 is a randomised, double-blind, placebo-controlled Phase 3 trial.

Adults with hereditary transthyretin amyloidosis (hATTR 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.
• Overall, based on the study submitted, additional benefit from diflunisal compared with the appropriate comparator therapy is not proven for adults with hereditary transthyretin amyloidosis with stage 1 or 2 polyneuropathy.
• In its dossier for the assessment of the additional benefit of diflunisal, the pharmaceutical manufacturer does not provide any data directly comparing the drug with the appropriate comparator therapy.
• The administration of placebo in the control arm of study H-23750 does not correspond to the appropriate comparator therapy.
• The study submitted is therefore not suitable for assessing additional benefit due to the lack of comparison with the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.