The CDE website shows that AstraZeneca's (AZN, Financial) antisense therapy Eplontersen (IONIS-TTR-LRx, trade name: Wainua) has applied for approval in China. It is intended to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
ATTRv-PN can cause peripheral nerve damage and mobility issues within five years of diagnosis, and untreated patients typically die within ten years. Globally, there are an estimated 30,000 to 50,000 cases of ATTR-CM, with around 40,000 patients suffering from ATTRv-PN.
Eplontersen (IONIS-TTR-LRx) is a ligand-conjugated antisense oligonucleotide (LICA) drug designed to reduce serum TTR production for treating both hereditary and non-hereditary ATTR. This drug was approved in the U.S. in December 2023. Under a global development and commercialization agreement, AstraZeneca and Ionis will jointly develop and commercialize Eplontersen in the U.S., while AstraZeneca will handle these activities in other regions, excluding Latin America.
