inotersen

Trade name: tegsedi

Nucleic acidapprovedOrphan Drug ^FDAFast Track ^FDA

Approved

Oct 5, 2018

Inotersen, sold under the brand name Tegsedi, is a 2'-O-(2-methoxyethyl) (2'-MOE) antisense oligonucleotide medication used for the treatment of nerve damage in adults with hereditary transthyretin-mediated amyloidosis. The sequence is TCTTG GTTACATGAA ATCCC, where C is methylated C, and the first and third section are MOE-modified. — Wikipedia

Clinical trial activity

4 trials · 4 clinical orgs · 2 marketing orgs

Phase 1

Phase 2

Phase 3

Phase 4

Earliest trial started Jan 1, 2016 (NCT02627820)

Timeline

2010s

Jul 24, 2012
Orphan Drug Designation
Jan 1, 2016
Earliest Phase 2 Sponsor(trial)
Oct 5, 2018
Ionis Pharmaceuticals, Inc. — Earliest FDA Approval
Oct 5, 2018
Akcea Therapeutics — NDA Secondary Org
Oct 5, 2018
Ionis Pharmaceuticals, Inc. — NDA Organization
Jan 1, 2019
Ionis Pharmaceuticals, Inc. — Earliest Phase 3 Sponsor(trial)

Indications

Approved for

Studied for

Mechanism of action

TransthyretinANTAGONIST
TTR antagonist
- Transthyretin (TTR) ↗
TransthyretinANTISENSE INHIBITOR
Transthyretin ANTISENSE inhibitor
Binds wild-type and mutant TTR mRNA which is subsequently degraded reducing TTR protein levels.
- Transthyretin (TTR) ↗
Transthyretin mRNAANTISENSE INHIBITOR
Transthyretin mRNA antisense inhibitor
Binds wild-type and mutant TTR mRNA which is subsequently degraded reducing TTR protein levels.

Approval history

approvedPriority reviewFast trackOct 5, 2018

Chemistry & pharmacology

Chirality: Single Stereoisomer
Inorganic: No
Polymer: No
Delivery: Parenteral
Availability: Discontinued
Multi-specific: No

Oral

Parenteral

Yes

Topical

Black box warningFirst in class

Sources

Also known as

gsk 299872
inotersen sodium
isis 420915
isis 420915

Clinical trials

NCT ID	Phase	Start date	Sponsor(s)
NCT04136184	Phase 3	Oct 31, 2019	Akcea Therapeutics, Ionis Pharmaceuticals, Inc.
NCT03702829	Phase 2	Dec 31, 2018	Harvard University
NCT02627820	Phase 2	Jan 1, 2016	GlaxoSmithKline, Harvard University, Ionis Pharmaceuticals, Inc.
NCT03400098	N/A	—	Ionis Pharmaceuticals, Inc.

Showing 4 of 4 trials

Page 1 / 1

Organizations

Research & Development (4)

Organization	Org type	Trials	As lead sponsor	Phases	Earliest year
Ionis Pharmaceuticals, Inc.	For profit	3	2	3	2016
Harvard University	Academic/Hospital	2	2	1	2016
Akcea Therapeutics	For profit	1	0	1	2019
GlaxoSmithKline	For profit	1	0	1	2016

4 organizations

Page 1 / 1

Marketing (2)

Organization	Org type	Relationship	Date
Akcea Therapeutics	For profit	NDA2	Oct 5, 2018
Ionis Pharmaceuticals, Inc.	For profit	NDA	Oct 5, 2018