Ionis and AstraZeneca have secured the European Commission (EC) approval for Wainzua (eplontersen) to treat hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults with stage one or stage two polyneuropathy.
ATTRv-PN is a debilitating disease that causes peripheral nerve damage and motor disability within five years of diagnosis. It can be fatal for the patient within a decade without treatment.
Wainzua is a once-monthly RNA-targeted medicine that suppresses transthyretin (TTR) production, targeting and reducing TTR protein production at its source in the liver.
AstraZeneca and Ionis are jointly developing and marketing Wainzua in the US as part of a global agreement and are seeking regulatory approval in other regions.
In December 2023, the drug received regulatory approval in the US, under the brand name Wainua, and is also approved in other countries, including Canada and the UK.
With the EC approval, Wainzua becomes the only approved medication in the European Union (EU) that can be self-administered monthly via an auto-injector.
Ionis CEO Brett Monia said: “Today’s 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.
“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.”
The EC approval follows the positive opinion of the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).
The approval is based on the Phase 3 NEURO-TTRansform study that evaluated the efficacy and safety of Wainzua in patients with ATTRv-PN at weeks 35, 66, and 85.
In the study, patients treated with Wainzua showed consistent benefits on co-primary endpoints, including serum TTR concentration and neuropathy impairment.
The neuropathy impairment using the modified Neuropathy Impairment Score +7 (mNIS+7).
Wainzua showed a favourable safety and tolerability profile throughout the trial.
Furthermore, the drug is also being evaluated in the global CARDIO-TTRansform Phase 3 study for treating adults with ATTR-CM, involving more than 1,400 patients.
