The FDA has granted accelerated approval to dostarlimab (Jemperli) for adults with stage II or III dMMR/MSI-H locally advanced rectal cancer — making it the first approved immunotherapy for this indication in the US. The decision was backed by the phase 2 AZUR-1 trial, where 47% of patients achieved a sustained clinical complete response at 12 months. This marks a major shift away from decades of reliance on chemotherapy, radiation, and surgery.
The FDA has granted accelerated approval to dostarlimab-gxly (Jemperli) for adult patients with stage II or III mismatch repair–deficient (dMMR) or microsatellite instability–high (MSI-H) locally advanced rectal cancer. This makes dostarlimab the first and only approved immunotherapy for this patient population in the US — a significant departure from the decades-long standard of chemotherapy, radiation, and surgery.
The approval was primarily supported by the phase 2 AZUR-1 trial (n = 154), in which patients received dostarlimab monotherapy at 500 mg IV every 3 weeks for 9 cycles. A second single-arm phase 2 trial (n = 50) provided additional supporting data, with patients achieving a 93% clinical complete response rate at 12 months and a 100% overall response rate.
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Why it matters: For patients with dMMR/MSI-H rectal cancer — a biomarker-defined subset — dostarlimab offers a personalized, biology-driven treatment option that could spare some patients from the significant morbidity associated with surgery and radiation, representing a meaningful step forward in precision oncology.
