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CHMP Recommends AstraZeneca and Ionis' Wainzua for EU Approval to Treat ATTRv-PN

NextEdge Admin

21 Oct 2024

2 min 19 sec

CHMP,Ionis’ Wainzua,RNA-targeted therapy

AstraZeneca 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 with polyneuropathy (ATTRv-PN) in adults. If approved, Wainzua will be the first EU-approved, self-administered, monthly treatment for ATTRv-PN via auto-injector.

The recommendation follows positive results from the Phase III NEURO-TTRansform trial, which demonstrated sustained benefits over 66 weeks. Wainzua improved co-primary outcomes, including serum transthyretin (TTR) concentration and neuropathy progression, measured by the modified Neuropathy Impairment Score +7 (mNIS+7). It also showed improvements in quality of life (QoL) on the Norfolk QoL-Diabetic Neuropathy Questionnaire, with favorable safety and tolerability.

ATTRv-PN is a debilitating, progressive disease that causes peripheral nerve damage and can become fatal within 10 years without treatment. Experts, including Dr. Laura Obici from the Amyloidosis Research Centre in Italy, emphasize the need for treatments that reduce TTR production to offer patients more time and improved QoL.

Wainzua, an RNA-targeted therapy, reduces TTR protein production at its source in the liver, aiming to treat all types of transthyretin amyloidosis. It was approved in the U.S. in December 2023 under the brand name Wainua, with AstraZeneca and Ionis commercializing it globally. Eplontersen also has Orphan Drug Designation in the U.S. and EU and is being evaluated in the ongoing CARDIO-TTRansform Phase III trial for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).