AstraZeneca Receives FDA Approval for Wainua in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

AstraZeneca and Ionis Pharmaceuticals today announced that the FDA has approved Wainua (eplontersen) for the treatment of adults with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Wainua is an antisense oligonucleotide (ASO) conjugated to a GalNAc ligand for enhanced liver delivery. In the Phase 3 NEURO-TTRansform trial, Wainua demonstrated a statistically significant improvement in the modified Neuropathy Impairment Score+7 (mNIS+7) compared to an external placebo comparator. The once-monthly subcutaneous injection offers a convenient dosing option for patients with this progressive, life-threatening disease.