GABA A receptor Inhibitors
23 drugsGABA A receptor Genetic Evidence Strong
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 100% directional consistency across 3 traits
- • Strong signal in nervous system disease, genetic, familial or congenital disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
13 totalGWAS and other genetic studies link GABRA1 to 13 diseases.
Loss-of-function causes disease; activation may help
Loss-of-function causes disease; activation may help
🔗 Colocalization Evidence 3 strong
max H4: 0.97eQTL/pQTL signals for GABRA1 colocalize with these GWAS traits, providing causal evidence that gene expression changes drive disease risk.
Understanding these scores
Association Score (0-1): Combines all evidence types (genetic, literature, drugs, animal models). Higher = more evidence linking target to disease. This is a ranking heuristic, not a confidence score.
L2G Score: Open Targets uses a machine learning model (Locus-to-Gene) to predict which gene is causal at each GWAS locus. L2G=0.5 means ~50% probability of being the causal gene. Only associations with L2G > 0.05 are included.
Odds Ratio (OR): From gene burden studies (UK Biobank, AstraZeneca PheWAS). Measures how loss-of-function variants affect disease risk. OR<1 = protective (inhibiting target may help), OR>1 = risk (losing function causes disease).
Beta (β): Effect size for continuous traits. β<0 = protective, β>0 = risk.
Clinical Translation (~1.8x): Based on Nelson et al. 2015: drug targets with genetic evidence have ~2x higher success rates in clinical trials. We estimate: Strong support (score ≥0.7) → ~1.8x, Moderate (0.3-0.7) → ~1.3x, Weak → baseline.
Colocalization (H4): Tests whether a GWAS signal and an eQTL/pQTL signal share the same causal variant. H4 is the posterior probability that both traits are associated AND share a causal variant. H4 > 0.8 = strong evidence that gene expression/protein levels drive disease risk. This links genetic variation → gene expression → disease, supporting the target-disease connection.
Top GABA A receptor Drugs
| Drug | Company | Approved | Indications |
|---|---|---|---|
| QUDEXY XR | UPSHER SMITH LABS | 2014 | 5 |
| EPRONTIA | AZURITY | 2021 | 5 |
| TROKENDI XR | SUPERNUS PHARMS | 2013 | 5 |
| TOPIRAMATE | Teva | 2003 | 4 |
| SYMPAZAN | COSETTE | 2018 | 2 |
| ZTALMY | IMMEDICA PHARMA | 2022 | 2 |
| ONFI | LUNDBECK PHARMS LLC | 2011 | 2 |
| VALTOCO | NEURELIS INC | 2020 | 2 |
| XANAX XR | UPJOHN | 2003 | 2 |
| TRIAZOLAM | ZYDUS PHARMS | 1994 | 1 |
| PROPOFOL | EMERGE BIOSCIENCE | 1999 | 1 |
| BUTALBITAL, ASPIRIN, CAFFEINE, AND CODEINE PHOSPHATE | NOVITIUM PHARMA | 1995 | 1 |
| EDLUAR | Viatris | 2009 | 1 |
| BYFAVO | ACACIA | 2020 | 1 |
| AMBIEN CR | COSETTE | 2005 | 1 |
| LOREEV XR | ALMATICA | 2021 | 1 |
| BUTALBITAL, ACETAMINOPHEN, CAFFEINE AND CODEINE PHOSPHATE | Hikma | 2001 | 1 |
| XCOPRI | SK LIFE | 2019 | 1 |
| AMBIEN | COSETTE | 1992 | 1 |
| HALCION | Pfizer | 1982 | 1 |
GABA A receptor Drug Modality Landscape
Modalities
Routes of Administration
GABA A receptor is amenable to small molecule drugs, with oral options available for convenient dosing.
GABA A receptor Clinical Trials 2,616 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on GABA A receptor. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 732 | 598 | 56 | 74 | 91% |
| Phase 2 | 461 | 298 | 77 | 82 | 79% |
| Phase 3 | 400 | 236 | 57 | 105 | 81% |
| Phase 4 | 1023 | 662 | 127 | 230 | 84% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Phase 3 Readout Calendar Pro
6 Phase 3 trials testing approved GABA A receptor drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting GABA A receptor. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
GABA A receptor Drug Approval Timeline (1981 - 2022)
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 20 companies competing
- • Market share by company
Full Drug Portfolio
- • All 23 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 23-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 1,085 (261 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on GABA A receptor. A trial counts if an approved drug acting on GABA A receptor is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.