AstraZeneca Plc. and Ionis Pharmaceuticals have received a positive recommendation for their medicine, Wainzua (eplontersen), from the European Union's Committee for Medicinal Products for Human Use (CHMP). This endorsement is for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, also known as hATTR-PN or ATTRv-PN.
Upon approval by the European Commission, Wainzua is set to be the sole EU-approved medication for ATTRv-PN that can be self-administered monthly using an auto-injector, according to AstraZeneca's statement.
The recommendation is rooted in the findings of the NEURO-TTRansform Phase III clinical trial, which indicated that Wainzua consistently and sustainably improved both neuropathy symptoms and patients' quality of life when compared to a placebo.
ATTRv-PN is a severe condition characterized by peripheral nerve damage, often resulting in motor disability within five years post-diagnosis and, if left untreated, is typically fatal within ten years.
In the United States, Wainzua was approved under the brand name Wainua for treating ATTRv-PN as of December 2023, with approvals expanding to multiple international markets.
Currently, eplontersen is undergoing assessment in the CARDIO-TTRansform Phase III trial, a significant study targeting transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This is the most extensive trial of its kind with over 1,400 participants involved.
