Eplontersen for hereditary transthyretin-mediated amyloid polyneuropathy

Eplontersen is currently in development for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). Hereditary transthyretin amyloidosis with polyneuropathy (formerly known as Familial Amyloid Polyneuropathy) is a rare disease caused by mutations in the gene that codes for transthyretin and is characterised by a multisystem extracellular deposition of amyloid (abnormal fibrous, extracellular, proteinaceous deposits found in organs and tissues), leading to the dysfunction of different organs and tissues. Therefore, hATTR-PN is a debilitating disease that can cause life-threatening situations.