Friday, FDA approved AstraZeneca Plc (NASDAQ: AZN) and Ionis Pharmaceuticals Inc's (NASDAQ: IONS) Wainua (eplontersen) for polyneuropathy (nerve damage) of hereditary transthyretin-mediated amyloidosis in adults (hATTR-PN or ATTRv-PN).
Wainua is the only approved medicine for ATTRv-PN that can be self-administered via an auto-injector.
The approval was based on the positive 35-week interim analysis from the NEURO-TTRansform Phase 3 trial, which showed that patients treated with Wainua demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) ...