givosiran

Trade name: Givlaari

Nucleic acidapprovedOrphan Drug ^FDABreakthrough Therapy ^FDA

Approved

Nov 20, 2019

Givosiran, sold under the brand name Givlaari, is a medication used for the treatment of adults with acute hepatic porphyria. Givosiran is a small interfering RNA (siRNA) directed towards delta-aminolevulinate synthase 1 (ALAS1), an important enzyme in the production of heme. — Wikipedia

Clinical trial activity

5 trials · 1 clinical orgs · 1 marketing orgs

Phase 1

Phase 2

Phase 3

Phase 4

Earliest trial started May 1, 2015 (NCT02452372)

Timeline

2010s

Jan 1, 2015
Alnylam Pharmaceuticals — Earliest Phase 1 Sponsor(trial)
Aug 29, 2016
Orphan Drug Designation
Jan 1, 2017
Alnylam Pharmaceuticals — Earliest Phase 3 Sponsor(trial)
Nov 20, 2019
Alnylam Pharmaceuticals — Earliest FDA Approval
Nov 20, 2019
Alnylam Pharmaceuticals — Marketing Organization
Nov 20, 2019
Alnylam Pharmaceuticals — NDA Organization

Indications

Approved for

Studied for

Mechanism of action

5-aminolevulinate synthase mRNADEGRADER
5-aminolevulinate synthase mRNA degrader
5-aminolevulinate synthase mRNARNAI INHIBITOR
5-aminolevulinate synthase mRNA RNAi inhibitor
The double stranded RNA interferes with aminolevulinate synthase 1 mRNA in the liver, leading to it's degradation and therefore reduces levels of toxic metabolites (aminolevulinic acid and porphobilinogen) that would otherwise be produced by functional ALAS1.

Approval history

approvedPriority reviewBreakthroughNov 20, 2019

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-as1

Clinical trials

NCT ID	Phase	Start date	Sponsor(s)
NCT03505853	Phase 1	Apr 26, 2018	Alnylam Pharmaceuticals
NCT03338816	Phase 3	Nov 1, 2017	Alnylam Pharmaceuticals
NCT02949830	Phase 1/Phase 2 (Phase 2)	Oct 1, 2016	Alnylam Pharmaceuticals
NCT02452372	Phase 1	May 1, 2015	Alnylam Pharmaceuticals
NCT04056481	N/A	—	Alnylam Pharmaceuticals

Showing 5 of 5 trials

Page 1 / 1

Organizations

Research & Development (1)

Organization	Org type	Trials	As lead sponsor	Phases	Earliest year
Alnylam Pharmaceuticals	For profit	5	5	4	2015

1 organizations

Page 1 / 1

Marketing (2)

Organization	Org type	Relationship	Date
Alnylam Pharmaceuticals	For profit	MKTG	Nov 20, 2019
Alnylam Pharmaceuticals	For profit	NDA	Nov 20, 2019