AstraZeneca PLC (LSE: AZN) (STO: AZN (Nasdaq: AZN) a global, science-led biopharmaceutical company, announced on Monday that Wainzua (eplontersen) has received a positive recommendation for approval from the Committee for Medicinal Products for Human Use (CHMP) in the European Union for treating adult patients with hereditary transthyretin-mediated amyloidosis (hATTR-PN). This recommendation is based on results from the NEURO-TTRansform Phase III trial, which demonstrated sustained benefits in neuropathy impairment and quality of life compared to placebo over 66 weeks.

If approved by the European Commission, Wainzua will be the only self-administered treatment in the EU for stage 1 and stage 2 hATTR-PN. The trial results highlighted significant improvements in serum transthyretin (TTR) concentrations and neuropathy impairment, alongside a favourable safety profile.

Wainzua is a once-monthly RNA-targeted medicine designed to reduce TTR protein production in the liver. It received US approval under the name Wainua in December 2023 and is now pursuing regulatory approvals in other regions, where AstraZeneca holds exclusive commercialisation rights outside the US. Eplontersen also holds Orphan Drug Designation in both the US and EU.

Currently, eplontersen is being evaluated in the CARDIO-TTRansform Phase III trial for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), which is the largest trial of its kind to date, involving over 1,400 participants. This treatment targets a critical area of unmet medical need, with an estimated 10,000 to 40,000 patients affected by hATTR-PN in Europe.

As part of a global development and commercialisation agreement, AstraZeneca and Ionis are commercialising Wainua for the treatment of ATTRv-PN in the US.