FDA grants GlaxoSmithKline's Jemperli accelerated approval in rectal cancer; many trial patients avoided surgery

Patient flow in AZUR-1 shows how the treatment was used. Of 154 treated patients, 110 (71%) received dostarlimab alone, had no additional treatment and did not progress; 26 (17%) underwent surgery; 13 (8%) received additional systemic therapy or radiation; one had progressive disease without further treatment; and four withdrew or were lost to follow-up. Among 15 patients who had surgery after dostarlimab alone because imaging suggested an incomplete response, 12 had a pathological complete response. Separately, FDA reviewed an investigator-led 50-patient trial in the same tumor type: cCR12 was 93% and the objective response rate 100%, with no patients receiving chemoradiation or rectal cancer surgery. Of 27 patients followed at least three years, one had a local recurrence treated with lymphadenectomy, one withdrew and one died of an unrelated cause.

The label recommends 500 mg every three weeks until disease progression or unacceptable toxicity, for up to nine cycles. It warns of immune-mediated adverse reactions, infusion-related reactions, complications of allogeneic hematopoietic stem cell transplantation and embryo-fetal toxicity. The review used the Real-Time Oncology Review and Assessment Aid programs and ran under Project Orbis with Health Canada, Israel's Ministry of Health, Swissmedic and the UK's Medicines and Healthcare products Regulatory Agency; those agencies' reviews are ongoing. FDA granted priority review and breakthrough therapy designation and approved the application four months ahead of its goal date. The application was part of the Commissioner's National Priority Review Voucher pilot.