AstraZeneca : Wainzua Recommended By CHMP For EU Approval In Hereditary Transthyretin Amyloidosis

(RTTNews) - AstraZeneca Plc. (AZN.L, AZN) and Ionis' Wainzua (eplontersen) has been recommended for approval by the Committee for Medicinal Products for Human Use (CHMP) in the European Union (EU) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN.

If approved by the European Commission, Wainzua will be the only approved medicine in the EU for the treatment of ATTRv-PN that can be self-administered monthly via an auto-injector, AstraZeneca said in a statement.

The recommendation is based on NEURO-TTRansform Phase III results, which showed that Wainzua demonstrated consistent and sustained benefits in improving neuropathy impairment and quality of life compared to placebo.

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 was approved under the brand name Wainua for the treatment of ATTRv-PN in the US in December 2023 and is now gaining approvals in additional countries worldwide.

Eplontersen is currently being evaluated in the CARDIO-TTRansform Phase III trial for treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), the largest of all ATTR-CM trials to date including over 1,400 participants.

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