Ionis' Eplontersen Receives FDA Orphan Drug Status For Misfolded Protein Disorder

The FDA has granted orphan drug designation to Ionis Pharmaceuticals Inc's 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). 
• It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR). 
• Related content: Benzinga's Full FDA Calendar.
• In December 2021, Ionis announced a strategic collaboration with AstraZeneca Plc AZN to develop and commercialize eplontersen.
• ATTR amyloidosis is caused by TTR amyloid deposits' inappropriate formation and aggregation in various tissues and organs. 
• Polyneuropathy due to hATTR and ATTR cardiomyopathy is caused by the accumulation of misfolded mutated TTR protein in the peripheral nerves and cardiac muscle, respectively.
• Price Action: IONS shares closed at $29.88 on Friday.