AstraZeneca and Ionis’ Wainua (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.1Wainua is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.2-4
The approval by the US Food and Drug Administration (FDA) was based on the positive 35-week interim analysis from the NEURO-TTRansform Phase III trial, 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).2,5 Positive results from the NEURO-TTRansform Phase III trial 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.2
Michael J. Polydefkis, M.D., Professor of Neurology at Johns Hopkins University School of Medicine and an investigator in the NEURO-TTRansform study, said: “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. 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.”
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.6Wainua is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of TTR protein at its source to treat both hereditary and non-hereditary forms of transthyretin-mediated amyloidosis (ATTR).3,7,8
Ruud Dobber, Executive Vice-President, BioPharmaceuticals Business Unit, AstraZeneca, said: “There is an urgent medical need for new therapies for people living with hereditary transthyretin-mediated amyloid polyneuropathy. The US approval of Wainua offers a new treatment option that provides consistent and sustained reduction in serum TTR concentration compared to baseline while halting disease progression and improving quality of life for people living with this debilitating condition.”
