Favourable opinion for reimbursement in the “treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy”.
Clinical Benefit
Moderate
The clinical benefit of WAINZUA 45 mg (eplontersen) solution for injection in pre-filled pen is moderate in the MA indication.
Clinical Added Value
no clinical added value
Considering:
evidence of the superiority of eplontersen versus placebo in NEURO-TTRansform, a randomised, open-label phase 3 trial, for the change in serum TTR concentration and the change in mNIS+7 score,
evidence of an improvement in quality of life, assessed by the Norfolk QOL-DN, quality of life score, as a co-primary endpoint in the NEURO-TTRansform study,
the design of the study and its methodological limitations, i.e. an indirect comparison of the eplontersen group with an external historic placebo group from the Neuro-TTR study, which was the pivotal study for inotersen [TEGSEDI], which reduces the scope of the efficacy results observed,
the absence of any available comparison versus a clinically relevant comparator, despite the fact that a direct comparison versus ONPATTRO (patisiran), TEGSEDI (inotersen) or VYNDAQEL (tafamidis) or a methodologically robust indirect study versus AMVUTTRA (vutrisiran) could have been feasible,
the limited duration of the study, of 18 months, which is insufficient to guarantee maintenance of efficacy in this chronic disease.
the safety profile of eplontersen that seems to be favourable, with follow-up limited to 18 months,
the Committee deems that WAINZUA 45 mg (eplontersen) solution for injection in pre-filled pen provides no clinical added value (CAV V) in the current care pathway, which includes therelevant comparators.
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