FDA grants accelerated approval to dostarlimab for locally advanced rectal cancer
Regulatory approval: On Oct 19, 2026, the FDA granted accelerated approval to dostarlimab (Jemperli; GSK) for adults with Stage II/III locally advanced rectal cancer that is MSI-H or dMMR, as determined by an FDA-authorized test. Approval was supported primarily by the single-arm AZUR-1 study, which reported a 47% sustained clinical complete response at 12 months (ICR) among 116 evaluable patients, and an ICR ORR of 79%. Safety includes immune-mediated toxicities and infusion reactions.
FDA accelerated approval: dostarlimab in MSI-H/dMMR locally advanced rectal cancer (Oct 19, 2026)
The FDA granted accelerated approval (Oct 19, 2026) to dostarlimab (Jemperli; GSK) for adults with Stage II/III locally advanced rectal cancer (LARC) that is microsatellite instability–high (MSI-H) or mismatch repair deficient (dMMR), as determined by an FDA-authorized test for solid tumors.
Supporting efficacy evidence
Efficacy was evaluated in AZUR-1 (NCT05723562), a multicenter, single-arm, open-label trial in 154 previously untreated patients with histologically confirmed MSI-H/dMMR Stage II/III LARC. Dostarlimab was given 500 mg IV every 3 weeks for nine cycles unless unacceptable toxicity or progression. Response assessment used endoscopy, rectal MRI, and CT.
Among 116 ICR-evaluable patients, cCR12 (sustained clinical complete response for 12 months) was 47% (95% CI 27–56), and ORR per ICR was 79% (95% CI 71–85). Median follow-up was 26.3 months; no progression was reported among patients with cCR12, cCR, or near CR at data cutoff.
FDA also reviewed investigator-led NCT04165772 (n=50): cCR12 93% (95% CI 82–99) and ORR 100% (95% CI 93–100).
Dosing and key safety warnings
Recommended dose: 500 mg every 3 weeks until progression or unacceptable toxicity, up to nine cycles. Label warnings include immune-mediated adverse reactions, infusion-related reactions, complications of allogeneic HSCT, and embryo-fetal toxicity.
Review used Project Orbis and RTOR; international partner reviews were ongoing at the time described.