Get all your news in one place.
100’s of premium titles.
One app.
Start reading
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.
Sign up to read this article
Read news from 100’s of titles, curated specifically for you.
Already a member? Sign in here
Related Stories
Top stories on inkl right now
One subscription that gives you access to news from hundreds of sites
Already a member? Sign in here
Our Picks
Fourteen days free
Download the app
One app. One membership.
100+ trusted global sources.