FDA approves GSK's Jemperli for a type of rectal cancer
The FDA has given fast-track approval to GSK's drug Jemperli for some people with rectal cancer that has spread nearby, Fierce Pharma and OncLive reported. It is the first drug of its kind for these patients. It may help some of them skip chemo, radiation and surgery.
The Food and Drug Administration has granted accelerated approval to GSK's immunotherapy Jemperli, or dostarlimab, for adults with stage 2 or 3 locally advanced rectal cancer that is mismatch repair deficient or microsatellite instability-high, Fierce Pharma and OncLive reported. These tumors have a specific genetic abnormality and make up 5% to 10% of rectal cancers, Fierce Pharma reported.
Monday's approval makes Jemperli the first immunotherapy for these patients, Fierce Pharma and OncLive reported. It offers an option that could help them avoid chemotherapy, radiation and life-altering surgery, Fierce Pharma reported.
The approval was based on the phase 2 AZUR-1 trial, OncLive reported. Among 116 patients evaluated by independent review, 47% had a sustained complete clinical response at 12 months, Fierce Pharma and OncLive reported. By investigators' assessment, the rate was 68%, OncLive reported. The FDA also reviewed an earlier single-arm trial of 50 patients, OncLive reported.
Andrea Cercek of Memorial Sloan Kettering Cancer Center, who led the study behind the approval, said about 30% of rectal cancer patients under standard care need a permanent colostomy, Fierce Pharma reported. "With just Jemperli alone, we were able to offer the majority of patients complete normal organ preservation, normal pelvic function," she said at a press briefing, according to Fierce Pharma. Five babies have been born to patients from her initial academic study, she said, Fierce Pharma reported.
None of the responders have seen their cancer return after a median follow-up of more than two years, according to Cercek and GSK, Fierce Pharma reported.
"For decades, treatment has relied on chemotherapy, radiation, and surgery," GSK chief scientific officer Tony Wood said in a release, OncLive reported.
Because the approval is accelerated, GSK must confirm the drug's benefit, Fierce Pharma reported. A phase 3 trial in colon cancer could serve that purpose and is expected to read out in 2028, GSK's Hesham Abdullah said, Fierce Pharma reported.
What we know: The FDA approved Jemperli for stage 2 or 3 rectal cancers with this genetic profile, based on response data.
What we don't: How long responses last for most patients, which confirmatory trials must show.
Where outlets agree Fierce Pharma and OncLive report that the FDA granted accelerated approval to Jemperli for stage 2 or 3 MSI-H or dMMR rectal cancer, with 47% of 116 evaluable patients in sustained complete response.
Where they differ The outlets cited here report the same key facts so far.