Rare nerve disease drug shows equal promise in men and women, trial finds
A new treatment for hereditary transthyretin amyloidosis—a rare genetic disorder that damages nerves—works comparably well in both sexes, according to analysis of a late-stage trial. The finding matters for drug developers and insurers weighing coverage decisions for the injectable therapy, which is already approved in multiple regions and addresses an unmet need in a small but critically ill patient population.
Originaltitel: Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy: an exploratory analysis of treatment effect in male and female patients
<p>Introduction/Aims: Eplontersen is approved in multiple regions for adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This exploratory analysis was conducted to evaluate the treatment effect of eplontersen by sex in patients with ATTRv-PN from the NEURO-TTRansform trial (NCT04136184).</p><p>Methods: Participants in NEURO-TTRansform who received ≥ 1 dose of eplontersen were included in this analysis; participants in the inotersen reference group who switched to eplontersen were excluded. Eplontersen was evaluated in the overall patient population and in a subgroup with cardiomyopathy. Change from baseline in the NEURO-TTRansform primary endpoints of serum transthyretin (TTR) levels to Week 65, modified Neuropathy Impairment Score +7 (mNIS+7) composite score to Week 66, and Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score to Week 66 were evaluated. A historical placebo group from the NEURO-TTR trial (NCT01737398) was included as a control.</p><p>Results: The eplontersen group comprised 100 (69.4%) male and 44 (30.6%) female patients. The placebo group comprised 41 (68.3%) male and 19 (31.7%) female patients. For both sexes, treatment with eplontersen decreased serum TTR levels by > 80% (vs. reductions of 3%–13% with placebo), maintained mNIS+7 scores, and improved Norfolk QoL-DN scores (vs. deterioration with placebo). The treatment effect of eplontersen was similar in male and female patients with cardiomyopathy.</p><p>Discussion: Eplontersen halted neuropathy impairment and improved quality of life versus placebo to a similar degree in both male and female patients with ATTRv-PN, including those with cardiomyopathy. These findings support the use of eplontersen as an effective treatment for ATTRv-PN regardless of sex.</p>