GABA-B Inhibitors
3 drugsAbout GABA-B
Gamma-aminobutyric acid type B receptors (GABA-B) are neuronal targets in the central nervous system activated by GABA. They play a crucial role in inhibitory neurotransmission, modulating neuronal excitability and synaptic transmission.
Human genetic studies provide strong validation for GABBR1 as a therapeutic target, with variants linked to neurodevelopmental disorder with language delay (score 0.77). Additional associations include global developmental delay and intellectual disability (score 0.62).
GABA-B is targeted by 3 FDA-approved small molecule drugs including XYWAV, XYREM and SODIUM OXYBATE. These drugs are approved for 3 therapeutic areas and marketed by companies including ASCENT PHARMS INC, JAZZ, and JAZZ PHARMS.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Cerebral Amyloid Angiopathy with only 1 trials.
Human Genetic Evidence Strong
GABBR1 has strong genetic support with a maximum score of 0.77 linking it to neurodevelopmental disorders.
Strong genetic support suggests a higher likelihood of success in clinical trials targeting GABBR1.
Evidence Across Diseases
8 totalGWAS and other genetic studies link GABBR1 to 8 diseases.
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 Drugs
The GABA-B drug market is concentrated among 3 companies, including ASCENT PHARMS INC and JAZZ PHARMS.
The limited number of players indicates relatively low competitive intensity, potentially easing market entry.
Drug Modality Landscape
Modalities
Routes of Administration
GABA-B is amenable to small molecule drugs, with oral options available for convenient dosing.
The exclusive use of small molecules suggests a whitespace opportunity for novel modalities like antibodies or peptides.
Clinical Trials 52 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 13 | 11 | 0 | 2 | 100% |
| Phase 2 | 20 | 10 | 3 | 6 | 77% |
| Phase 3 | 7 | 5 | 1 | 1 | 83% |
| Phase 4 | 12 | 8 | 2 | 2 | 80% |
Top Sponsors
By Modality
Top Conditions
Drug Approval Timeline (2002 - 2022)
The first GABA-B targeting drug was approved in 2002 (XYREM), with the most recent approval in 2020 (XYWAV).
The approval timeline indicates continued interest in GABA-B as a drug target, but may be approaching saturation.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 3 companies competing
- • Market share by company
Full Drug Portfolio
- • All 3 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 3-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Competition: High (15 sponsors)
- • White space: 10 underexplored indications
- • Success rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Calculate These Metrics
Target Attractiveness Score
A 0-100 score based on trial activity, sponsor diversity, and completion rates. Calculated from 32 clinical trials targeting GABA-B.
Completion rate: Percentage of trials that reached their planned endpoint. Trials terminated early, withdrawn, or suspended are not counted—these often indicate safety issues, lack of efficacy, or strategic pivots.
- Highly Attractive (80+): High trial activity, many sponsors, strong completion rates
- Attractive (60-79): Good trial activity and validation
- Moderate (40-59): Moderate interest from sponsors
- Low (under 40): Limited trial activity or validation concerns
Strategic Insights
Auto-generated insights based on trial analytics including competition intensity, white space opportunities, modality shifts, and failure patterns. We analyze trial sponsors, phases, indications, and outcomes.
Risk Signals
- High Competition: Many sponsors competing for this target (may reduce market opportunity)
- High Failure Risk: Low trial completion rates suggest development challenges
- Low Validation: Limited trial activity or poor outcomes indicate uncertain viability
- White Space Available: Underexplored indications present opportunities