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WAINUA™ (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis
U.S. FDA approval based on Phase 3 NEURO-TTRansform results showing WAINUA demonstrated consistent and sustained benefit halting neuropathy disease
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"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 Additional regulatory reviews for WAINUA underway in rest of world WAINUA will be available in the U.S. in January 2024CARLSBAD, Calif., Dec. 21, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today 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.\n\n \n \n \n \n \n \n\n \nThe approval was based on the positive 35-week interim analysis from the Phase 3 NEURO-TTRansform study which showed patients treated with WAINUA demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7), and key secondary endpoint of quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). Positive results from the Phase 3 NEURO-TTRansform study were published in The Journal of the American Medical Association (JAMA) further demonstrating the benefit of WAINUA across the spectrum of ATTRv-PN at 35, 66 and 85-weeks.\n\"Many people living with hereditary transthyretin-mediated amyloid polyneuropathy are unable to fully enjoy their lives because of the relentless, progressive and debilitating effects of the disease,\" said Michael J. Polydefkis, M.D., professor of neurology at Johns Hopkins University School of Medicine and an investigator in the NEURO-TTRansform study. \"Approval of WAINUA represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease.\"\nATTRv-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...