EMA Recommends Nod of Ionis & AstraZeneca's Wainzua to treat polyneuropathy

Ionis Pharmaceuticals, Inc. revealed that the Committee for Medicinal Products for Human Use (CHMP) in the European Union (EU) has recommended the approval of Ionis' and AstraZeneca's Wainzua (eplontersen) for treating hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, also known as hATTR-PN or ATTRv-PN. If Wainzua is given the green light by the European Commission, it will be the sole authorized medication in the EU for treating ATTRv-PN that can be self-administered once a month using an auto-injector.

The CHMP relied on the favorable results of the NEURO-TTRansform phase 3 clinical trial to form its opinion. The trial revealed that over 66 weeks, patients who received Wainzua consistently and enduringly benefited in terms of serum transthyretin (TTR) concentration and neuropathy impairment evaluated by modified Neuropathy Impairment Score +7 (mNIS+7), as well as quality of life (QoL) measured on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) compared to an external placebo. Wainzua maintained a positive safety and tolerability record during the NEURO-TTRansform trial.

ATTRv-PN is a severe illness that results in harm to the peripheral nerves causing motor impairment within five years of being diagnosed and, if left untreated, typically results in death within ten years. Wainzua is a medication that targets RNA and is meant to decrease the creation of TTR protein directly.