 The FDA has granted orphan drug designation to Ionis Pharmaceuticals Incs NASDAQ: IONS eplontersen, an investigational antisense medicine for transthyretin-mediated amyloidosis. Eplontersen is currently in Phase 3 trials for amyloid transthyretin cardiomyopathy ATTR-CM and amyloid transthyretin polyneuropathy ATTR-PN. 