patisiran

Trade name: ONPATTRO

Nucleic acidapprovedOrphan Drug ^FDAFast Track ^FDABreakthrough Therapy ^FDA

Approved

Aug 10, 2018

Patisiran, sold under the brand name Onpattro, is a medication used for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis, a fatal rare disease that is estimated to affect 50,000 people worldwide. — Wikipedia

Clinical trial activity

12 trials · 2 clinical orgs · 1 marketing orgs

Phase 1

Phase 2

Phase 3

Phase 4

Earliest trial started Mar 1, 2012 (NCT01559077)

Timeline

2010s

Jan 1, 2012
Alnylam Pharmaceuticals — Earliest Phase 1 Sponsor(trial)
Jan 1, 2012
Alnylam Pharmaceuticals — Earliest Phase 2 Sponsor(trial)
Jun 14, 2012
Orphan Drug Designation
Jan 1, 2013
Alnylam Pharmaceuticals — Earliest Phase 3 Sponsor(trial)
Aug 10, 2018
Alnylam Pharmaceuticals — Earliest FDA Approval
Aug 10, 2018
Alnylam Pharmaceuticals — NDA Secondary Org
Aug 10, 2018
Alnylam Pharmaceuticals — NDA Organization

Indications

Approved for

Studied for

Mechanism of action

TransthyretinANTISENSE INHIBITOR
TTR antisense inhibitor
- Transthyretin (TTR) ↗
Transthyretin mRNARNAI INHIBITOR
Transthyretin mRNA RNAi inhibitor
Short interfering RNA (siRNA) which binds the 3-UTR region of wild-type and mutant transthyretin mRNA leading to degradation and reduced TTR protein levels.

Approval history

approvedPriority reviewFast trackBreakthroughAug 10, 2018

Chemistry & pharmacology

Chirality: Single Stereoisomer
Inorganic: No
Polymer: No
Availability: Prescription Only
Multi-specific: No

Oral

Parenteral

Topical

First in class

Sources

Also known as

aln-ttr02
aln-ttr02
patisiran
patisiran
patisiran sodium

Clinical trials

NCT ID	Phase	Start date	Sponsor(s)
NCT05023889	Early Phase 1 (Phase 1)	Oct 31, 2021	Alnylam Pharmaceuticals, Austin Neuromuscular Center
NCT03997383	Phase 3	Aug 31, 2019	Alnylam Pharmaceuticals
NCT03862807	Phase 3	Mar 27, 2019	Alnylam Pharmaceuticals
NCT03759379	Phase 3	Jan 18, 2019	Alnylam Pharmaceuticals
NCT02939820	N/A	Oct 18, 2016	Alnylam Pharmaceuticals
NCT02510261	Phase 3	Jul 1, 2015	Alnylam Pharmaceuticals
NCT02053454	Phase 1	Jan 1, 2014	Alnylam Pharmaceuticals
NCT01960348	Phase 3	Nov 1, 2013	Alnylam Pharmaceuticals
NCT01961921	Phase 2	Oct 1, 2013	Alnylam Pharmaceuticals
NCT01617967	Phase 2	May 1, 2012	Alnylam Pharmaceuticals
NCT01559077	Phase 1	Mar 1, 2012	Alnylam Pharmaceuticals
NCT05505838	N/A	—	Alnylam Pharmaceuticals

Showing 12 of 12 trials

Page 1 / 1

Organizations

Research & Development (2)

Organization	Org type	Trials	As lead sponsor	Phases	Earliest year
Alnylam Pharmaceuticals	For profit	12	11	4	2012
Austin Neuromuscular Center	Academic/Hospital	1	1	1	2021

2 organizations

Page 1 / 1

Marketing (4)

Organization	Org type	Relationship	Date
Alnylam Pharmaceuticals	For profit	NDA2	Aug 10, 2018
Alnylam Pharmaceuticals	For profit	MKTG	—
Alnylam Pharmaceuticals	For profit	IND	—
Alnylam Pharmaceuticals	For profit	NDA	Aug 10, 2018