Rectal Cancer Drug Dostarlimab Achieves 100% Remission, Gets FDA Breakthrough Therapy Status

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to the immunotherapy drug Dostarlimab (Jemperli) following a clinical trial that achieved a 100% remission rate in patients with a specific type of rectal cancer.

The designation, announced in December 2024, shows the drug’s potential as a transformative treatment for mismatch repair–deficient (dMMR) rectal cancer, possibly expediting its regulatory review.

"Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s)," the agency explained.

The FDA’s decision comes after a landmark study conducted by Memorial Sloan Kettering Cancer Center (MSKCC), where all 18 participants with locally advanced dMMR rectal cancer experienced complete tumor disappearance without chemotherapy, radiation, or surgery. The trial’s findings were hailed as a historic milestone, marking the first time a cancer drug has led to complete remission in every patient.

Dostarlimab, a PD-1 checkpoint inhibitor, works by helping the immune system recognize and attack cancer cells. The unprecedented success of the trial has positioned the drug as a potential non-invasive alternative to traditional cancer treatments. The FDA Breakthrough Therapy designation is reserved for treatments that show substantial improvements over existing therapies for serious conditions, allowing for faster development and review.

"This new treatment is also proving very durable. Most people on the trial have been free of cancer for at least a year, and the original participants have been healthy for up to four years and counting. The success rate remains 100%," explained Andrea Cercek, MD, MSK gastrointestinal oncologist and clinical trial leader.

With the FDA’s support, larger clinical trials will now be conducted to evaluate the long-term effectiveness of Dostarlimab. If future studies confirm its success, the drug could redefine cancer treatment, offering a highly effective alternative for patients with dMMR colorectal cancer and potentially other cancers.

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