AstraZeneca & Ionis Wainua Bags US FDA Nod To Treat Polyneuropathy

Wainua (eplontersen) from AstraZeneca and Ionis has been licensed in the United States for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, also known as hATTR-PN or ATTRv-PN. Wainua is the only licensed drug for the treatment of ATTRv-PN that may be delivered by self-injector.

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), as per pharmabiz.

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 severe disease that causes peripheral nerve degeneration and motor impairment within five years of diagnosis and is often fatal within a decade if not treated.6 Wainua is a ligand-conjugated antisense oligonucleotide (LICA) drug that is intended to diminish TTR protein production at its source in order to treat both hereditary and non-hereditary forms of transthyretin-mediated amyloidosis (ATTR).