Biotechnology company Ionis Pharmaceuticals, Inc. (Nasdaq:IONS) announced on Monday that Ionis' and AstraZeneca's WAINZUA (eplontersen) has received a positive recommendation from the European Union's Committee for Medicinal Products for Human Use (CHMP) for treating hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN) in adults.

If approved by the European Commission, WAINZUA will be the only self-administered monthly treatment available in the EU for this condition.

The recommendation follows favourable results from the NEURO-TTRansform Phase 3 trial, where WAINZUA showed sustained improvements in TTR levels, neuropathy impairment, and quality of life. WAINZUA also demonstrated a favorable safety and tolerability profile throughout the trial. ATTRv-PN causes motor disability within five years and is often fatal within a decade without treatment. WAINZUA is designed to target RNA and reduce TTR protein production.