Drug updated on 10/21/2024
Dosage Form | Injection (subcutaneous; 45 mg/0.8 mL) |
Drug Class | Transthyretin-directed antisense oligonucleotide |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Latest News
Summary
- This summary is based on the review of one randomized controlled trial(s). [1]
- Eplontersen treatment led to a significant reduction in serum transthyretin concentration, with an adjusted mean percentage reduction of -81.7% at week 65, compared to -11.2% for the placebo group. The difference between the groups was -70.4% (95% CI, -75.2% to -65.7%; P < .001).
- Neuropathy impairment, measured by the mNIS+7 composite score, showed an adjusted mean change of 0.3 for eplontersen from baseline to week 66, compared to 25.1 for placebo, resulting in a difference of -24.8 (95% CI, -31.0 to -18.6; P < .001).
- In terms of quality of life (Norfolk QoL-DN Total Score), eplontersen showed an adjusted mean change of -5.5 at week 66, compared to 14.2 for placebo, with a difference of -19.7 (95% CI, -25.6 to -13.8; P < .001).
- Adverse events leading to study drug discontinuation occurred in 6 patients (4%) in the eplontersen group and 2 patients (3%) in the placebo group.
- Two deaths were reported in the eplontersen group due to cardiac arrhythmia and intracerebral hemorrhage, both attributed to disease-related sequelae, while no deaths were reported in the placebo group.
- The study included 168 adults with ATTRv polyneuropathy (Coutinho stage 1 or 2), with 144 patients in the eplontersen group (mean age 53.0 years; 69% male) and 60 patients in the placebo group (mean age 59.5 years; 68% male). No significant differences in effectiveness or safety outcomes were reported between different population types or subgroups.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Wainua (eplontersen) Prescribing Information. | 2023 | AstraZeneca Pharmaceuticals LP, Wilmington, DE |
Randomized Controlled Trials
Document Title | Sex Distribution | Year | Source |
---|---|---|---|
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy. | 204Subjects F: 31% M: 69% | 2023 | JAMA |
Sex Distribution:
F:31%
M:69%
204Subjects
Year:
2023
Source:JAMA