AstraZeneca-Ionis Pharma Wainzua approved in EU for hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy

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 3 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.

“THe approval of WAINZUA in Europe offers adults with hereditary transthyretin-mediated amyloidosis with polyneuropathy a new, self-administered treatment option that provides consistent suppression of transthyretin production and improves neuropathy impairment and quality of life,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “With approvals in North America, UK and now across the EU, we are proud of the continued progress as we and our partner, AstraZeneca, rapidly and effectively deliver WAINZUA to people around the world.”

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.