Use of the approved therapy Wainua (eplontersen) effectively slows disease progression and improves quality of life among adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) — characterized by damage to the peripheral nerves found outside the brain and spinal cord — across a range of disease-causing mutations.
These findings, which researchers called “suggestive of consistent benefits,” come from an exploratory analysis of NEURO-TTRansform (NCT04136184), a now-completed large clinical trial that supported the approval of Wainua as the first treatment for hATTR-PN that can be self-administered by patients via an autoinjector.
The global Phase 3 trial, sponsored by Ionis Pharmaceuticals, had tested the effectiveness of the injection therapy in people with hATTR-PN with a documented genetic mutation.