FDA grants accelerated approval to dostarlimab monotherapy for dMMR/MSI-H rectal cancer
The FDA granted accelerated approval on October 19 to single-agent dostarlimab for adults with locally advanced dMMR/MSI-H rectal cancer after 71% of trial patients avoided surgery, radiation and chemotherapy.
On October 19, the Food and Drug Administration granted accelerated approval to dostarlimab-gxly (Jemperli, GSK) as a standalone first-line treatment for adults with Stage II or III locally advanced rectal cancer whose tumors are mismatch repair-deficient (dMMR) or microsatellite instability-high (MSI-H), as determined by an FDA-authorized test. [1][2]
Approval rested on the single-arm AZUR-1 trial (NCT05723562) of 154 previously untreated patients given nine intravenous doses over roughly six months. With a median follow-up of 26.3 months, 110 of 154 patients (71.4%) remained on non-operative management after dostarlimab alone without surgery, pelvic radiation, chemotherapy or disease progression. Twenty-six patients (17%) underwent surgery, and of 15 operated on for suspected incomplete response, 12 showed pathological complete response upon resection. [1][3]
The scope boundary is essential for patients and clinicians: dMMR or MSI-H defects occur in only about 3% of locally advanced rectal cancers. Because accelerated approval rests on single-arm response durability rather than randomized long-term survival data, close non-operative surveillance—including endoscopy and MRI at Week 6, Week 12, and every four months for two years—is required. [1][2][4]
Take it further
Read the FDA clinical review summary and monitoring schedule For oncology teams and patients. Details FDA-authorized dMMR/MSI-H testing eligibility and the Week 6, Week 12 and four-month non-operative surveillance protocol.
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- FDA · Technical approval summary and AZUR-1 data
Primary October 19 CDER / Oncology Center of Excellence regulatory summary with complete AZUR-1 disposition (N=154) and ICR/investigator response rates.
- FDA · October 19 press announcement
Official announcement noting the ~3% dMMR/MSI-H prevalence in locally advanced rectal cancer and non-operative monitoring requirements.
- ClinicalTrials.gov · AZUR-1 protocol (NCT05723562)
Trial registry record for the multicenter open-label Phase 2 study.
- AJMC · Independent clinical reporting
October 19 clinical coverage corroborating the accelerated approval and trial endpoints.
Images & figures
In AZUR-1, 110 of 154 patients (71%) avoided surgery, radiation, and chemotherapy with no progression at cutoff.
Euangelion · original data figure · Source · CC BY 4.0 · Download figure