Givlaari

(Givosiran)

ApprovedRNA therapy

Givlaari is a rna therapy that works as ALAS1 Sirna.[2] It is being used across 2 indications, with its most advanced program in Approved.[1]

Givlaari is one of 4 sirna programs targeting Primary Hyperoxaluria. It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Primary HyperoxaluriaActive, not recruiting

Hereditary Transthyretin AmyloidosisApproved

Primary HyperoxaluriaApproved

Active, not recruiting

Hereditary Transthyretin AmyloidosisApproved

Approved

Competition

Competitive Landscape

3 competitors in Primary Hyperoxaluria

#DrugCompanyPhaseTrials

Givlaari

Alnylam Pharmaceuticals

Approved

5 2

DCR-PHXC

DDicerna Pharmaceuticals, Inc., a Novo Nordisk company

Phase 3

2 3

YOLT-203

RRenJi Hospital

Phase 1

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Clinical

Trials by Indication[1]

Acute Intermittent Porphyria3 trials

A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 1·NCT03505853·10 enrolled

A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 1/2·NCT02949830·16 enrolled

A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 1·NCT02452372·40 enrolled

Acute Hepatic Porphyria2 trials

Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

Approved for marketing· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals··NCT04056481

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

Completed· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 3·NCT03338816·94 enrolled

Regulatory

FDA Approval Timeline[4]

Nov 20, 2019

GIVLAARI

NDA NDA212194

Nov 20, 2019

GIVLAARI

NDA NDA212194

Oct 28, 2021

GIVLAARI

NDA NDA212194

Feb 10, 2023

GIVLAARI

NDA NDA212194

Apr 29, 2024

GIVLAARI

NDA NDA212194

Sources

References

[1]
ClinicalTrials.gov — 5 trial records for Givosiran. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Givosiran compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Givosiran. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA212194). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Givlaari. U.S. Food & Drug Administration.
[6]
PubMed — 1 publication indexed for Givosiran. U.S. National Library of Medicine.
[7]
SEC EDGAR — Alnylam Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: ALAS1 Sirna[2]
Modality: RNA therapy[2]
Highest Phase: Approved[1]
Company: Alnylam Pharmaceuticals

Patent Landscape[5]

Patent Cliff

Aug 24, 2028

Earliest patent expiration — generic entry possible after this date

Patent #Expires

10131907Aug 24, 2028

8828956Dec 4, 2028

8106022Dec 12, 2029

9631193Mar 15, 2033

10125364Mar 15, 2033

3 more patents

9133461Nov 30, 2033

11028392Oct 3, 2034

10119143Oct 3, 2034

Key Publications[6]

Givosiran in Acute Hepatic Porphyria: ENVISION Long-Term

N Engl J MedNov 10, 2022DOI: 10.1056/NEJMoa2215643

Givlaari

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Acute Intermittent Porphyria3 trials

Acute Hepatic Porphyria2 trials

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

Key Publications[6]

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