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Wainua (EPLONTERSEN)
Wainua works by binding to the transthyretin RNA to prevent the production of the transthyretin protein.
Wainua (eplontersen) is an antisense oligonucleotide developed by Astrazeneca Ab, targeting the transthyretin protein. It is classified as a Transthyretin-directed RNA Interaction [EPC] and has been approved by the FDA in 2025 for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis. Wainua has a half-life of 3 weeks and is currently patented, with no generic manufacturers available. It is administered to interact with the transthyretin RNA, reducing the production of the transthyretin protein that causes the disease. Key safety considerations include the potential for infusion reactions and liver enzyme elevations.
At a glance
| Generic name | EPLONTERSEN |
|---|---|
| Sponsor | Astrazeneca Ab |
| Drug class | Transthyretin-directed RNA Interaction [EPC] |
| Modality | Oligonucleotide |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2025 |
Mechanism of action
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.
Approved indications
- Polyneuropathy of hereditary transthyretin-mediated amyloidosis
Common side effects
- Vitamin A decreased
- Vomiting
- Proteinuria
- Injection site reactions
- Blurred vision
- Cataract
Key clinical trials
- Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy (PHASE2,PHASE3)
- Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
- The Eplontersen Pregnancy and Lactation Outcomes Study
- EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy (PHASE3)
- An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) (PHASE3)
- A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (PHASE3)
- CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study
- The CARDIO-TTRansform Scintigraphy Sub-study
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Wainua CI brief — competitive landscape report
- Wainua updates RSS · CI watch RSS
- Astrazeneca Ab portfolio CI