The European Union's Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of AstraZeneca and Ionis Pharmaceuticals’ Wainzua (eplontersen) to treat hereditary transthyretin-mediated amyloidosis in adults with stage one or two polyneuropathy (ATTRv-PN).
ATTRv-PN is a progressive disease that causes peripheral nerve damage and motor disability, typically leading to death within a decade if untreated.
Wainzua's potential approval by the European Commission would make it the only self-administered, monthly treatment via an auto-injector for ATTRv-PN in the EU.
AstraZeneca and Ionis Pharmaceuticals are collaborating on the commercialisation of Wainzua following a global agreement established in December 2021. AstraZeneca holds exclusive commercialisation and development rights in regions outside the US.
AstraZeneca bioPharmaceuticals business unit executive vice-president Ruud Dobber stated: “Due to the progressive nature of polyneuropathy of hereditary transthyretin-mediated amyloidosis, it is critical to have timely diagnosis and new therapies to help people have greater control over this potentially fatal disease.
“Today’s recommendation brings Wainzua one step closer for patients in Europe, and if approved, will offer a new treatment option that can provide consistent TTR [transthyretin] suppression and results in improved quality of life.”
The CHMP's recommendation is based on results from the NEURO-TTRansform Phase III trial, which demonstrated consistent and sustained benefits in patients treated with Wainzua over 66 weeks.
The trial focused on co-primary endpoints of serum TTR concentration and neuropathy impairment, along with a key secondary endpoint assessing the quality of life.
Wainzua's safety and tolerability were also favourably noted throughout the trial.
It works as a once-monthly silencer that suppresses serum TTR production at its source in the liver and is designed to treat all kinds of ATTR.
The US Food and Drug Administration approved Wainzua for ATTRv-PN treatment in December 2023 under the brand name Wainua, and additional global approvals are being sought.
Eplontersen has received orphan drug designation in both the US and the EU to treat ATTR.
