Oncology · 3 h ago
FDA approves dostarlimab for selected locally advanced rectal cancers
The FDA granted accelerated approval for dostarlimab (Jemperli) as monotherapy for adults with previously untreated stage II or III dMMR or MSI-H locally advanced rectal cancer. The decision was based on interim results from the 154-patient phase 2 AZUR-1 study; longer follow-up is needed to confirm durable clinical benefit.
- FDA approval covers biomarker-selected stage II or III disease.
- AZUR-1 included 154 patients; follow-up remains ongoing.
- Nearly three-fourths had not needed standard treatments at analysis.
- Accelerated approval requires confirmation of lasting clinical benefit.
The FDA approved dostarlimab (Jemperli) for adults with previously untreated stage II or III locally advanced rectal cancer whose tumors are mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H), confirmed by an FDA-authorized test. The immune checkpoint inhibitor targets PD-1. These biomarker-defined tumors are estimated to account for about 3% of locally advanced rectal cancers.
The decision was based on interim results from AZUR-1 (NCT05723562), a phase 2 study of 154 patients treated with up to nine doses over six months. After a median follow-up of more than two years, almost three-fourths had not required surgery, radiotherapy or chemotherapy. An earlier 50-patient study (NCT04165772) reported no detectable cancer after treatment; 93% maintained that status for at least 12 months.
The accelerated approval may offer eligible patients an alternative to treatments that can affect bowel, urinary and sexual function, but the FDA requires continued follow-up to establish lasting benefit and assess recurrence. Dostarlimab can cause severe or fatal immune-mediated reactions; commonly reported side effects included fatigue, rash, diarrhea and itching. The approval was granted to GlaxoSmithKline.
In practice
For adults with previously untreated stage II or III locally advanced rectal cancer that is dMMR or MSI-H by an FDA-authorized test, dostarlimab monotherapy is now an FDA-approved option that may allow some patients to avoid chemotherapy, radiotherapy and surgery. In the 154-patient phase 2 AZUR-1 study, almost three-fourths had not required these treatments after more than two years’ median follow-up. This is accelerated approval based on interim evidence; longer follow-up is required to confirm durability and recurrence outcomes. Immune-mediated toxicity can be severe or fatal.
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U.S. Food and Drug Administration: FDA Approves Immunotherapy That May Allow Certain Patients with Rectal Cancer to Avoid Chemotherapy, Radiation and Surgery ↗This is an automated AI-condensed summary that has not yet been reviewed by an editor. Always consult the full item at the original source.