FDA Grants Accelerated Approval to Dostarlimab for Rectal Cancer
The FDA has granted accelerated approval to dostarlimab for adults with stage II or III locally advanced rectal cancer that is MSI-H or dMMR, a defined molecular group.
The Food and Drug Administration has granted a dostarlimab rectal cancer approval to dostarlimab, sold as Jemperli, for adults with stage II or III locally advanced rectal cancer that is MSI-H or dMMR. The decision, dated October 19, covers a group of patients whose tumors have specific molecular features: microsatellite instability-high or mismatch repair deficient disease. For many of these patients, standard care has involved surgery and radiation, treatments that can carry lasting effects on bowel, bladder, and sexual function. An immunotherapy option that can shrink tumors without those steps is therefore a significant change in how care may be planned.
Dostarlimab is a PD-1 immunotherapy, a type of medicine that helps the immune system recognize and respond to cancer cells. In the trial supporting the approval, most patients were treated with immunotherapy alone, and the FDA reported a sustained clinical complete response at 12 months of 47 percent in 116 patients evaluable by independent central review. That figure describes patients whose cancer could no longer be detected by the measures used in the study, a result that, for some, may allow surgery and radiation to be avoided or deferred while monitoring continues.
What the dostarlimab rectal cancer approval means
Accelerated approval is not the end of the evidence trail. The approval was granted four months ahead of the FDA goal date, under priority review and a national priority review voucher pilot program, and it carries a confirmatory trial requirement. The confirmatory study, AZUR-1, is a Phase 2 study that is active and not recruiting, with a projected completion date of July 31, 2030, for the requirement. If confirmatory evidence does not verify benefit, the approval can be revisited, so patients should understand both the promise and the limits of the current data.
The approval applies only to the defined molecular group, not to all rectal cancer, and eligibility depends on testing for MSI-H or dMMR status. Patients should discuss testing, monitoring, possible side effects of immunotherapy, and the plan if the tumor does not respond fully, with their oncology team. For the patients who do qualify, the decision adds a treatment path that was not available before, and it reflects a broader shift toward matching cancer therapy to tumor biology rather than to location alone.
Source: The Priority Review
