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Inotersen · Tegsedi, ISIS-420915

Oligonucleotide (Antisense oligonucleotide) · Gene silencing: transthyretin (TTR) · developed by Akcea Theraps

Approved FDA, Oct 2018

1 Registered trials

What is Inotersen?

Inotersen is an antisense oligonucleotide targeting transthyretin (TTR) mRNA. It binds to TTR mRNA in liver cells, inhibiting translation of both wild-type and mutant TTR protein, thereby lowering TTR blood levels and reducing amyloid deposits. It is studied in hereditary transthyretin-mediated amyloid polyneuropathy.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: Tegsedi, ISIS-420915, GSK-2998728, TEGSEDI
Drug type: Oligonucleotide (Antisense oligonucleotide)
Targets: transthyretin (TTR)
Mechanism: Gene silencing: transthyretin (TTR)
Pathways: protein synthesis inhibition
Therapy area: Neurology
Developer: Akcea Theraps
In trials of: IONS
FDA status: Approved as Tegsedi (October 5, 2018), NDA211172 · FDA label
UNII: 0IEO0F56LV

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT04136184	Phase 3	Hereditary Transthyretin-Mediated Amyloid Polyneuropathy	Completed	IONS

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Other drugs acting on TTR

Patisiran FDA approved Vutrisiran FDA approved ALN-TTRSC04 Phase 1 NTLA-2001 Phase 3 Nucresiran Phase 3

Sources

Updated Oct 3, 2026.

Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z