The investigational agent eplontersen halted neuropathy disease progression and improved neuropathy impairment and quality of life for patients with hereditary transthyretin-mediated amyloid polyneuropathy in a pivotal phase 3 trial. AANAM AAN2023
The investigational agent eplontersen halted neuropathy disease progression and improved neuropathy impairment and quality of life for patients with hereditary transthyretin-mediated amyloid polyneuropathy in a pivotal phase 3 trial.
Khella reported final data from the NEURO-TTRansform trial April 24 at the American Academy of Neurology 2023 Annual Meeting.ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within 5 years of diagnosis. Without treatment, it is generally fatal within a decade.
Eplontersen led to a significant mean reduction of 82% in TTR serum concentration compared to an 11% reduction with placebo . Treatment with eplontersen also significantly improved patient-reported quality of life, as assessed by the validated Norfolk Quality of Life Questionnaire–Overall, 58% of patients who were treated with eplontersen showed improvements in quality of life at 66 weeks, compared with 20% of patients treated with placebo.
"Long-term safety and tolerability data are currently being assessed in the open-label extension," Khella said.
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