SV2A Inhibitors
6 drugsAbout SV2A
Synaptic Vesicle Glycoprotein 2A (SV2A) is a transmembrane protein in the brain that regulates neurotransmitter release. Its role in neurotransmitter release makes it a compelling target for therapeutic intervention.
Human genetic studies provide strong validation for SV2A as a therapeutic target (max score 0.84). Genetic variants are linked to skeletal abnormalities (score 0.84), developmental and epileptic encephalopathy (score 0.76), and type 2 diabetes (score 0.58), suggesting activation may be beneficial.
SV2A is targeted by 6 FDA-approved small molecule drugs, including LEVETIRACETAM (KEPPRA) and BRIVIACT. These drugs are used for CNS disorders and other therapeutic areas.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Brain Tumors with only 2 trials.
SV2A Genetic Evidence Strong
SV2A has strong genetic support with a max score of 0.84 across 13 diseases.
Strong genetic support suggests SV2A-targeting drugs have a higher probability of clinical success.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 100% directional consistency across 1 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 SV2A to 13 diseases.
Loss-of-function causes disease; activation may help
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for SV2A 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 SV2A Drugs
Four companies have approved drugs targeting SV2A, including ZYDUS PHARMS USA INC and UCB INC.
The competitive landscape indicates moderate market concentration, suggesting potential entry barriers.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| BRIVIACT | UCB INC | 2016 | 1 |
| BRIVARACETAM | Aurobindo Pharma | 2022 | 1 |
| KEPPRA XR | UCB INC | 2008 | 1 |
SV2A Drug Modality Landscape
Modalities
Routes of Administration
SV2A is amenable to small molecule drugs, with oral options available for convenient dosing.
The modality landscape suggests an opportunity to develop non-small molecule drugs targeting SV2A.
SV2A Clinical Trials 178 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 32 | 26 | 3 | 1 | 90% |
| Phase 2 | 51 | 28 | 13 | 10 | 68% |
| Phase 3 | 54 | 30 | 8 | 16 | 79% |
| Phase 4 | 41 | 24 | 8 | 6 | 75% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Phase 3 Readout Calendar Pro
2 Phase 3 trials testing approved SV2A drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting SV2A. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
SV2A Drug Approval Timeline (1999 - 2022)
The first SV2A-targeting drug was approved in 1999 (KEPPRA), with the most recent in 2022 (BRIVARACETAM).
The approval timeline indicates continued interest in SV2A as a drug target.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 4 companies competing
- • Market share by company
Full Drug Portfolio
- • All 6 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 6-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 173 clinical trials targeting SV2A.
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