Eplontersen sodiumProduct ingredient for Eplontersen
- Name
- Eplontersen sodium
- Drug Entry
- Eplontersen
Eplontersen is a transthyretin-directed antisense oligonucleotide with 3 covalently linked e N-acetyl galactosamine residues for hepatic delivery. It was previously investigated as a potential treatment for hereditary transthyretin-mediated amyloidosis.2 Hereditary transthyretin-mediated amyloidosis is caused primarily by pathogenic variants of the TTR gene, leading to the formation and thus accumulation of insoluble and misfolded amyloid in multiple organs, although wild-type misfolded TTR has also been observed to contribute to the disease pathophysiology.3 As eplontersen targets both the WT and mutant TTR, it poses tremendous promises as a treatment.4
On December 21, 2023, the FDA approved eplontersen under the brand name WAINUA for the treatment of adult polyneuropathy of hereditary transthyretin-mediated amyloidosis. This approval was based on positive results demonstrated in the 35-week interim analysis from the Phase 3 NEURO-TTRansform study, where a consistent improvement in the Neuropathy Impairment Score +7 (mNIS+7) and the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) Score were observed.6
- Accession Number
- DBSALT003492
- Structure
- Synonyms
- Not Available
- UNII
- WSP2DHR2BD
- CAS Number
- Not Available
- External Links
- Wikipedia
- Eplontersen
- Predicted Properties
- Not Available
