Friday, July 10, 2026

Drug profile · INN

eplontersen

eplontersen (WAINZUA) is a transthyretin antisense inhibitor.

EMA: approved Nervous system (N07) N07XX21
Patents linked 0
Pipeline programs 3
News articles 1
Drug details — eplontersen

Quick answer

eplontersen (WAINZUA) is a transthyretin antisense inhibitor.

Key facts

INN eplontersen
Brand WAINZUA
Mechanism Transthyretin antisense inhibitor
Target Transthyretin mRNA
Therapeutic class Nervous system (N07)
ATC code N07XX21

Mechanism of action

Transthyretin antisense inhibitor

Primary target: Transthyretin mRNA

Approval history

Authority Status Date
EMA approved

Recent coverage

Pipeline programs

  1. Eplontersen Phase 3 · IONIS PHARMACEUTICALS · Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
  2. Eplontersen Phase 3 · IONIS PHARMACEUTICALS · Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
  3. Eplontersen Phase 1 · IONIS PHARMACEUTICALS · Healthy Participants

Related & competitor drugs

  1. aripiprazole Nervous system (N05)
  2. brexpiprazole Nervous system (N05)
  3. brivaracetam Nervous system (N03)
  4. bupivacaine Nervous system (N01)
  5. buprenorphine Nervous system (N07)
  6. caffeine citrate Nervous system (N06)

Frequently asked questions

What is eplontersen?

eplontersen (WAINZUA) is a transthyretin antisense inhibitor.

What is the brand name for eplontersen?

WAINZUA is a marketed brand name for eplontersen. Check FDA Drugs@FDA for the current US label holder and prescribing information.