AstraZeneca / Ionis’ Wainua approved in U.S. for treatment of polyneuropathy

AstraZeneca (AZN) and Ionis (IONS) has been approved in the U.S. for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.1 WAINUA is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector. The approval by the US Food and Drug Administration 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 concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 , and key secondary endpoint of quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy. Positive results from the NEURO-TTRansform Phase III trial were published in The Journal of the American Medical Association further demonstrating the benefit of WAINUA across the spectrum of ATTRv-PN at 35, 66 and 85 weeks.