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PrWAINUA™ eplontersen injection
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WAINUA® Important Safety Information

Indications and clinical use:

WAINUA (eplontersen injection) is indicated for the treatment of polyneuropathy associated with stage 1 or stage 2 hereditary transthyretin-mediated amyloidosis in adults.

Relevant warnings and precautions:

  • Reduced serum vitamin A levels and recommended supplementation
  • Driving and operating machinery
  • Potential risk of ocular symptoms
  • Use in pregnant or breastfeeding women
  • Risks related to reproductive health, including teratogenic risk

For more information:

Please consult the Product Monograph for important information relating to adverse reactions, drug interactions, and dosing information, which has not been discussed in this piece.

The Product Monograph is also available by calling 1-877-404-8277 or emailing ask-medical@astrazeneca.ca.

Reference: WAINUA® Product Monograph. AstraZeneca Canada Inc. August 26, 2025.

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WAINUA®

TTR Suppression Data*

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  • Trial Design
  • Efficacy Data
  • Safety & Tolerability Profile

WAINUA reduced serum TTR concentrations as early as Week 5, with a significant difference in the change from baseline vs. external placebo at Week 35 (when steady state was reached) and through Week 65 (p<0.001; pharmacodynamic endpoint)1,2

Change in serum TTR concentration through 65 weeks1,2†

Change in serum TTR concentration through 65 weeks1,2Change in serum TTR concentration through 65 weeks1,2
  • LSM % change from baseline at Week 35 (interim analysis): 81.1% WAINUA vs. -14.5% external placebo (difference: -66.6%; p<0.001)
  • LSM % change from baseline at Week 65 (final analysis): -81.6% WAINUA vs. -11.2% external placebo (difference: -70.4%; 95% CI: -75.2, -65.7; p<0.001)
  • Adapted from the WAINUA Product Monograph and Coelho T, et al.1,2
  • † Following baseline, external placebo was assessed at Weeks 5, 8, 13, 23, 35, 47, 59, and 65, while WAINUA was assessed at Weeks 5, 9, 13, 25, 35, 49, 57, and 65.2

TTR reduction sustained up to Week 85 with WAINUA.1

See NEURO-TTRansform trial design

TTR: transthyretin; LSM: least squares mean; CI: confidence interval.

* Clinical significance unknown.

References: 1. WAINUA® Product Monograph. AstraZeneca Canada Inc. August 26, 2025. 2. Coelho T, Marques Jr W, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy. JAMA. 2023;330(15):1448-1458.

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WAINUA® is a registered trademark of AstraZeneca AB, used under license by AstraZeneca Canada Inc.

The AstraZeneca logo is a registered trademark of AstraZeneca AB, used under license by AstraZeneca Canada Inc.

© AstraZeneca Canada Inc. 2025

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