fitusiran

Nucleic acidapprovedOrphan Drug FDA

Approved

Mar 28, 2025

Clinical trial activity

11 trials · 4 clinical orgs · 1 marketing orgs

Phase 1
4
Phase 2
8
Phase 3
7
Phase 4
0

Earliest trial started Jan 1, 2014 (NCT02035605)

Timeline

2010s

  1. Aug 12, 2013

    Orphan Drug Designation

  2. Jan 1, 2014

    Earliest Phase 1 Sponsor(trial)

  3. Jan 1, 2018

    Genzyme — Earliest Phase 3 Sponsor(trial)

  4. Jan 1, 2019

    Genzyme — Earliest Phase 2 Sponsor(trial)

2020s

  1. Mar 28, 2025

    Earliest FDA Approval

  2. Mar 28, 2025

    Genzyme — Marketing Organization

Indications

Approved for

Mechanism of action

Approval history

  • approvedMar 28, 2025

Chemistry & pharmacology

Inorganic
No
Polymer
No
Multi-specific
No

Oral

No

Parenteral

No

Topical

No

Sources

Also known as

  • aln-at3sc
  • fitusiran
  • fitusiran

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT07285460Phase 3Dec 19, 2025Sanofi
NCT06145373Phase 1Dec 15, 2023Sanofi
NCT05662319Phase 3Jan 13, 2023Sanofi
NCT04394286Phase 1/Phase 2 (Phase 2)May 30, 2020Shire
NCT03974113Phase 2/Phase 3 (Phase 3)Jun 17, 2019Genzyme, Sanofi
NCT03754790Phase 3Jan 9, 2019Genzyme, Sanofi
NCT03417245Phase 3Jul 2, 2018Genzyme, Sanofi
NCT03549871Phase 3May 28, 2018Sanofi
NCT03417102Phase 3Mar 22, 2018Genzyme, Sanofi
NCT02554773Phase 1/Phase 2 (Phase 2)Sep 1, 2015Alnylam Pharmaceuticals
NCT02035605Phase 1Jan 1, 2014Alnylam Pharmaceuticals
Showing 11 of 11 trials
Page 1 / 1

Organizations

Research & Development (4)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
SanofiFor profit8422018
GenzymeFor profit4412018
Alnylam PharmaceuticalsFor profit2222014
ShireFor profit1112020
4 organizations
Page 1 / 1

Marketing (1)

OrganizationOrg typeRelationshipDate
GenzymeFor profitMKTGMar 28, 2025