AstraZeneca Receives FDA Approval for Wainua in Hereditary Transthyretin-Mediated Amyloidosis

AstraZeneca today announced that the FDA has approved Wainua (eplontersen) for the treatment of adults with hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN). Eplontersen is a ligand-conjugated antisense oligonucleotide that demonstrated a statistically significant improvement in neuropathy impairment score and quality of life in the Phase 3 NEURO-TTRansform trial. The monthly subcutaneous self-administration offers a convenient treatment option for patients with this rare progressive disease. This approval expands the treatment options available for hereditary ATTR amyloidosis.