Biomarker and systemic therapy

HLA-A*02:01 and tebentafusp in uveal melanoma

HLA-A*02:01 is a true treatment-defining biomarker: a positive result opens the tebentafusp pathway in unresectable or metastatic UM.

For patients and clinicians

Phase III evidence: 1-year overall survival 73% vs 59%, median OS 21.7 vs 16.0 months; hazard ratio for death 0.51. Nathan P et al. N Engl J Med. 2021.Tap the image to open the full-size vector infographic.

What to know

Tebentafusp is a TCR-bispecific molecule that recognises a gp100 peptide presented by HLA-A*02:01 and redirects T cells toward the tumour cell.

Test HLA firstWithout HLA-A*02:01 this branch does not open.

OS is the key endpointPhase III and 3-year follow-up demonstrated an OS advantage.

RECIST is not the whole storyRadiographic shrinkage does not fully capture benefit.

For clinicians

Three-year analysis: median OS 21.6 versus 16.9 months; 3-year survival 27% versus 18% in the control arm. Early adverse events require step-up dosing and monitoring.

Long-term update · 2026

The survival advantage persists at 5 years

16%alive at 5 years
tebentafusp

8%alive at 5 years
control

21.6months median OS
vs 16.9 months

HR 0.6795% CI 0.54–0.85

The final 5-year phase III analysis confirmed a durable overall-survival advantage. This complements the pivotal 1-year comparison shown above.

5-year follow-up — PubMed

Key references

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