EU Approves Wainzua for Amyloidosis

Wainzua is the only approved medicine in the EU for the treatment of ATTRv-PN that can be self-administered monthly via an auto-injector.

The approval by the European Commission (EC) follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP). The approval is based on the positive NEURO-TTRansform Phase III trial which showed that through 66 weeks, patients treated with Wainzua 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) versus external placebo. Wainzua continued to demonstrate a favorable safety and tolerability profile throughout the NEURO-TTRansform trial.

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. Wainzua  is a once-monthly RNA-targeted medicine designed to reduce the production of TTR protein at its source.

Wainzua was approved under the brand name Wainua for the treatment of ATTRv-PN in the U.S. in December 2023 and has gained approvals in additional countries worldwide, including Canada and the United Kingdom. As part of a global development and commercialization agreement, AstraZeneca and Ionis are jointly developing and commercializing Wainua in the U.S. The companies are seeking regulatory approval in other parts of the world where AstraZeneca has exclusive rest of world commercialization and development rights.