PharmaRadar360

PharmaRadar360

NeurologyDrug intelligence

Wainua

Eplontersen

TTR-directed antisense oligonucleotide (GalNAc conjugate). Developed by AstraZeneca. Clinical, regulatory, and source evidence are connected below.

Company
AstraZeneca
Target / MOA
TTR mRNA
Aliases
Wainua 路 Wainzua
Signals
15
Clinical trials
11
Approvals
0
Evidence sources
15

Latest signals

What changed

The three most recent source-backed changes linked to this drug.

clinical trialClinicalTrials.gov

Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations

N/A 路 RECRUITING 路 AstraZeneca

clinical trialClinicalTrials.gov

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Phase 3 路 COMPLETED 路 Ionis Pharmaceuticals, Inc.

clinical trialClinicalTrials.gov

The Eplontersen Pregnancy and Lactation Outcomes Study

N/A 路 RECRUITING 路 AstraZeneca

Approved uses

What regulators have approved

The indications section of each current US label, word for word, and the EU summary of each authorised product. Official sources as of Oct 21, 2026.

United States (FDA label)

Wainua 路 NDA217388 路 label version 20, effective Apr 15, 2026 路 DailyMed

Label section 1

1 INDICATIONS AND USAGE WAINUA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

European Union (EMA)

Wainzua 路 EMEA/H/C/006295 路 Authorised, authorised Mar 6, 2025 (Commission decision C(2025) 1557) 路 EPAR

Treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy.

EMA's summary; the full indication is in the product information on the EPAR page.

Regulatory history

Approvals over time

US original approvals and efficacy supplements (new indications, populations or dosing) from Drugs@FDA; EU authorisations and CHMP opinions on extensions. Official sources as of Oct 21, 2026.

United States

NDA217388 路 ASTRAZENECA AB 路 first approved Dec 21, 2023

1 efficacy supplement approved

European Union

  • Wainzua: Authorised since Mar 6, 2025

Patents and exclusivity

What FDA lists (US)

Patents and exclusivity as FDA lists them in the Orange Book (drugs) and the Purple Book (biologics), with approved generics and biosimilars. Expired listings are not shown. Official sources as of Oct 21, 2026.

NDA217388 (Orange Book)

3 listed patents, latest expiry Aug 25, 2034
  • US 9127276 路 expires May 1, 2034 路 drug substance
  • US 9181549 路 expires May 1, 2034 路 drug substance
  • US 10683499 路 expires Aug 25, 2034 路 drug substance, drug product, U-2378

Exclusivity: NCE until Dec 21, 2028 (some products only: WAINUA (AUTOINJECTOR) EQ 45MG BASE/0.8ML (EQ 45MG BASE/0.8ML)); ODE-461 until Dec 21, 2030 (some products only: WAINUA (AUTOINJECTOR) EQ 45MG BASE/0.8ML (EQ 45MG BASE/0.8ML)) 路 Orange Book entry

Generics and biosimilars

The Orange Book lists no approved generic (ANDA).

FDA's listings only: an approved generic or licensed biosimilar may not be on the market yet. Not a forecast of when they may launch, and not legal advice.

Safety

Boxed warnings and FDA safety communications

The boxed warning on each current US label, and FDA Drug Safety Communications that name the drug (FDA's list from January 2020). Official sources as of Oct 21, 2026.

Boxed warning

The current US label has no boxed warning.

FDA Drug Safety Communications

None on FDA's list names this drug.

Clinical trials

Key active studies

A focused view of recently updated studies 路 11 linked records.

N/ARECRUITINGNCT07654855

Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations

AstraZeneca 路 Updated Sep 14, 2026

Phase 3COMPLETEDNCT04136171

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Ionis Pharmaceuticals, Inc. 路 Updated Sep 3, 2026

N/ARECRUITINGNCT07205666

The Eplontersen Pregnancy and Lactation Outcomes Study

AstraZeneca 路 Updated Aug 27, 2026

Phase 2ACTIVE NOT RECRUITINGNCT07608354

A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM

AstraZeneca 路 Updated Jul 14, 2026

Phase 3ACTIVE NOT RECRUITINGNCT06194825

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

AstraZeneca 路 Updated Jun 9, 2026

Phase 3ENROLLING BY INVITATIONNCT05667493

An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Ionis Pharmaceuticals, Inc. 路 Updated Apr 17, 2026

Regulatory timeline

Approvals and agency activity

Verified regulatory records in reverse chronological order.

No regulatory event is available in this snapshot.

Source evidence

Where this profile comes from

Facts link back to original registries, regulators, labels, and publishers. Counts change as source records update.

ClinicalTrials.govofficial 路 Latest Sep 23, 202615
Professional referenceThis profile summarizes linked public evidence. It is not medical, legal, regulatory, or investment advice. Verify decisions against original sources.