The FDA has granted accelerated approval to dostarlimab (Jemperli) as the first immunotherapy for adults with stage II/III MSI-H/dMMR locally advanced rectal cancer. Backed by the AZUR-1 trial, the drug showed a 68% sustained clinical complete response rate at 1 year. This marks a potential shift away from the traditional chemotherapy, radiation, and surgery approach that often impacts patients' quality of life.
The FDA has granted accelerated approval to dostarlimab (Jemperli, GlaxoSmithKline), making it the first and only immunotherapy approved for adults with stage II or III microsatellite instability-high (MSI-H) or mismatch repair–deficient (dMMR) locally advanced rectal cancer. The PD-1 antagonist offers a biomarker-driven alternative to the current standard of care — chemotherapy, radiation, and surgery — treatments that can significantly affect quality of life, including risks of sexual dysfunction and infertility.
The approval was primarily supported by the phase 2 AZUR-1 trial, in which patients received 500 mg of dostarlimab IV every 3 weeks for up to nine cycles. Additional supporting data came from a separate investigator-led phase 2 trial of 50 similar patients, which showed even stronger results.
Why it matters: For decades, rectal cancer treatment has relied on aggressive multimodal therapy with lasting side effects. This approval opens the door to a personalized, biology-driven approach for a defined subset of patients — potentially sparing them from surgery and its long-term consequences.
