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: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.
