US FDA Approves AstraZeneca and Ionis' Wainua for Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

AstraZeneca and Ionis Pharmaceuticals announced on December 21, 2023, that the U.S. Food and Drug Administration (FDA) has approved Wainua (eplontersen). The approval is for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. This marks the first regulatory approval in the U.S. for Wainua and positions it as the only self-administered treatment option for ATTRv-PN available via auto-injector.

The FDA's decision was based on positive results from the NEURO-TTRansform Phase III trial. The study demonstrated that patients treated with Wainua experienced consistent and sustained improvements in serum transthyretin (TTR) concentration and neuropathy impairment, measured by the modified Neuropathy Impairment Score +7 (mNIS+7). Key secondary endpoints, including quality of life measured by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), also showed significant benefit.

Hereditary transthyretin-mediated amyloid polyneuropathy (ATTR-PN) is a progressive and debilitating condition that leads to severe nerve damage. Without treatment, it is typically fatal within a decade of diagnosis. Wainua, a ligand-conjugated antisense oligonucleotide (LICA) medicine, works by reducing the production of the TTR protein at its source.

AstraZeneca and Ionis are co-commercializing Wainua in the U.S. and are seeking regulatory approval in Europe and other international markets. The drug has received Orphan Drug Designation in both the U.S. and the EU, indicating its potential to address unmet medical needs in rare diseases. Wainua is expected to be available in the U.S. in January 2024.