FDA to Review Eplontersen for ATTR Polyneuropathy

The Food and Drug Administration (FDA) has accepted for review the New Drug Application for eplontersen for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).

Hereditary ATTR is a severe, progressive and life-threatening disease caused by the abnormal formation and aggregations of transthyretin (TTR) amyloid deposits in various tissues and organs. Eplontersen is an investigational ligand-conjugated antisense medicine designed to reduce the production of TTR protein.

The submission includes data from the phase 3 NEURO-TTRansform study, which compared the efficacy and safety of eplontersen to the historical placebo arm from the Tegsedi^® (inotersen) NEURO-TTR trial (ClinicalTrials.gov Identifier: NCT01737398).

Findings from the 35-week interim analysis showed that treatment with eplontersen led to a significant mean reduction in serum TTR concentration, as well as a significant treatment effect on the modified Neuropathy Impairment Score +7 (a measure of neuropathic disease progression), compared with the historical placebo arm (both P <.0001). Moreover, treatment with eplontersen improved patient-reported quality of life, as measured by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (key secondary endpoint; P <.0001).

“Overall, the interim analysis demonstrated eplontersen has the potential to make a positive impact on disease progression and improve quality of life in a substantial number of patients,” said Eugene Schneider, MD, executive vice president and chief clinical development officer at Ionis.

The FDA has assigned a Prescription Drug User Fee Act action date of December 22, 2023 to the application.

Eplontersen is also being evaluated in the phase 3 CARDIO-TTRansform study (ClinicalTrials.gov Identifier: NCT04136171) for transthyretin amyloid cardiomyopathy.

This article originally appeared on MPR