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.
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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 (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).
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.
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 to treat both hereditary and non-hereditary forms of transthyretin-mediated amyloidosis (ATTR).
As part of a global development and commercialisation agreement, AstraZeneca and Ionis will commercialise Wainua for the treatment of ATTRv-PN in the US and are seeking regulatory approval in Europe and other parts of the world.
This agreement was recently expanded to include exclusive rights for AstraZeneca to commercialise Wainua in Latin America in addition to all other countries outside the US.
Wainua was granted Orphan Drug Designation in the US and in the EU for the treatment of ATTR.
Wainua will be available in the US in January 2024. ■