Tafamidis (1) – Vyndaqel®

• Clinical trials
  • The assessment of additional benefit is based on the minor effect on neurological impairment identified in the registration trial, as measured using the ‘Neuropathic Impairment Score of the Lower Limb’ (NIS-LL) scale.
  • The study population for the registration trial comparing tafamidis meglumine with placebo comprised 128 patients in the ‘safety population’, 125 in the intention-to-treat population, and 87 patients who were followed up until the end of the study (18 months) without any protocol violations.

Adult patients with hereditary transthyretin amyloidosis (hATTR) at stage 1 of polyneuropathy

• There is a minor additional benefit.
• The G-BA notes that the patient population included in the registration trial exhibits only minor symptoms.
• The mean modified body mass index (mBMI) at study enrolment was 1008, which is a major increase compared to the prognostically significant value of 600.
• The registration trial included only patients with the V30M mutation, meaning that the effect on other mutations was not investigated.
• Morbidity – Neurological impairment (NIS-LL scale)
  • The assessment of additional benefit is based on the minor effect on neurological impairment identified in the registration trial, as measured using the ‘Neuropathic Impairment Score of the Lower Limb’ (NIS-LL) scale.
  • The mean score of 9.9 points recorded at study enrolment corresponds to only 11 per cent of the maximum possible score of 88 points on the NIS-LL scale for measuring neurological impairment.
• Morbidity – Modified body mass index (mBMI)
  • The trend in mBMI, measured as a secondary endpoint in the study, showed a significant difference with more favourable values for the group treated with tafamidis; however, the G-BA considers the clinical relevance of the effects observed in the study to be questionable.

Courtesy translation only, please refer to the German original.