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Benzinga
Benzinga
Technology
Vandana Singh

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

The FDA has granted orphan drug designation to Ionis Pharmaceuticals Inc's (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). 
  • 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 (NASDAQ:AZNto 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.
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