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Drug

Givosiran

Approved · EMA
Class5-aminolevulinate synthase mRNA RNAi inhibitorEmerging researchLate-stage development

Also known as Givlaari.

RxNorm2265712ChEMBLCHEMBL4297760ATCA16AX16UNIIROV204583W
1
Research papers
1
Protein targets
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias

Clinical trial2024-04-22Results posted · ClinicalTrials.gov

A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1

Clinical trial2024-03-12Results posted · ClinicalTrials.gov

Therapeutic siRNA: state of the art.

Research2020-06-19Signal transduction and targeted therapy

Approval: Givlaari (EMA)

Regulatory2020-03-02EMA

A Drug-Drug Interaction Study to Investigate the Effect of Givosiran on the Pharmacokinetics (PK) of Midazolam, Caffeine, Losartan, Omeprazole, and Dextromethorphan in Patients With Acute Intermittent Porphyria (AIP) Who Are Asymptomatic High Excreters (ASHE)

Clinical trial2019-01-10Completed · ClinicalTrials.gov

A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)

Clinical trial2017-09-06Completed · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Givosiran
Aliases & brands
Givlaari
RxNorm CUI
2265712
ChEMBL ID
CHEMBL4297760
ATC codes
A16AX16
UNII
ROV204583W
Primary mechanism
5-aminolevulinate synthase mRNA RNAi inhibitor
Regulatory jurisdictions
ema

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

ENSG00000023330
ENSG00000023330
RNAI INHIBITOR5-aminolevulinate synthase mRNA RNAi inhibitor

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2020-03-02
Latest approval
2020-03-02
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2020-03-02Approval
Approval: Givlaari (EMA)

Indication: Treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

4 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
4
registered trials across all phases
LATEST COMPLETION 2021
1
Late-stage (III+)
4
Completed
PHASE DISTRIBUTIONn = 4
Phase 12Phase 1 / 21Phase 31

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias

CompletedPH IIIAlnylam Pharmaceuticalsn=942021

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1

CompletedPH I/IIAlnylam Pharmaceuticalsn=162021

ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias

CompletedPH IIIAlnylam Pharmaceuticalsn=942021

A Drug-Drug Interaction Study to Investigate the Effect of Givosiran on the Pharmacokinetics (PK) of Midazolam, Caffeine, Losartan, Omeprazole, and Dextromethorphan in Patients With Acute Intermittent Porphyria (AIP) Who Are Asymptomatic High Excreters (ASHE)

CompletedPH IAlnylam Pharmaceuticalsn=102019

A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)

CompletedPH IAlnylam Pharmaceuticalsn=402017

Research activity

1 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Most influential

Therapeutic siRNA: state of the art.

Signal transduction and targeted therapy · 2020 · 991 cites
Recent papers

Therapeutic siRNA: state of the art.

Signal transduction and targeted therapy · 2020 · 991 cites
Journals, researchers & institutions
Top journals
  • Signal transduction and targeted therapy1
Leading researchers
  • Hu B1
  • Huang Y1
  • Liang XJ1
  • Peng L1
  • Weng Y1
  • Zhao Y1
  • Zhong L1
Leading institutions
free-text, unnormalised
  • Aix-Marseille University1
  • Chinese Academy of Sciences (CAS)1
  • National Center for International Biotargeting Theranostics1
  • School of Life Science1

Related drugs

2 matches

Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).

Acetylgalactosamine

Studied across the same disease area as Givosiran in the shared literature.

1 shared disease1 shared paper
Patisiran

Studied across the same disease area as Givosiran in the shared literature.

1 shared disease1 shared paper
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.