FDA Approves Wainua (eplontersen) for the Treatment of Adults with Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis
Ionis Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has approved Ionis and AstraZeneca’s Wainua™ (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. Wainua is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.
U.S. FDA approval based on Phase 3 NEURO-TTRansform results showing Wainua demonstrated consistent and sustained benefit halting neuropathy disease progression and improving neuropathy impairment and quality of life.
ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. Wainua is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of TTR protein at its source. Wainua will be available in the U.S. in January 2024. Additional regulatory reviews for Wainua are underway in the rest of world.
