Currently Enrolling Trials
Wainua (eplontersen) is a transthyretin-directed antisense oligonucleotide.
Wainua is specifically indicated for the treatment of the polyneuropathy of hereditary transthyretin mediated amyloidosis in adults.
- Wainua is supplied as a solution for subcutaneous injection.
- The recommended dosage is 45 mg administered by subcutaneous injection once monthly.
- Missed Dose: Administer Wainua as soon as possible after a missed dose. Resume dosing at monthly intervals from the date of the most recently administered dose.
Mechanism of Action
Wainua (eplontersen) is an antisense oligonucleotide-GalNAc conjugate that causes degradation of mutant and wild-type TTR mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.
Adverse effects associated with the use of Wainua may include, but are not limited to, the following:
- vitamin A decreased
Clinical Trial Results
The FDA approval of Wainua was based on positive 35-week interim analysis from the NEURO-TTRansform Phase 3 trial.
The trial enrolled adult patients with ATTRv-PN Stage 1 or Stage 2 compared to the external placebo group from the Teggsedi (inotersen) NEURO-TTR registrational trial that Ionis completed in 2017. The comparison of efficacy and safety for Wainua
versus external placebo was based on data up to week 66, and all patients were followed on treatment until week 85, when they had the option to transition into an open-label extension study