Drug Profile
Eplontersen - AstraZeneca/Ionis Pharmaceuticals
Alternative Names: AKCEA-TTR-LRx; Eplontersen sodium - AstraZeneca/Ionis Pharmaceuticals; ION-682884; ION-TTR-LRx; IONIS-TTR-LRx; WAINUALatest Information Update: 29 Feb 2024
Price :
$50
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At a glance
- Originator Ionis Pharmaceuticals
- Developer Akcea Therapeutics; AstraZeneca; Ionis Pharmaceuticals
- Class Amides; Amino sugars; Antisense oligonucleotides; Drug conjugates
- Mechanism of Action Gene silencing; Prealbumin expression inhibitors
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Marketed Transthyretin-related hereditary amyloidosis
Most Recent Events
- 08 Feb 2024 Eplontersen - AstraZeneca/Ionis Pharmaceuticals receives Fast Track designation for Transthyretin-related hereditary amyloidosis [SC] (In adults) in USA
- 08 Feb 2024 Ionis and AstraZeneca seek regulatory approval for eplontersen in Europe and other parts of the world for transthyretin-related hereditary amyloidosis
- 22 Jan 2024 Launched for Transthyretin-related hereditary amyloidosis (In adults) in USA (SC)