FDA Approves Immunotherapy That May Let Some Rectal Cancer Patients Skip Treatments

The Food and Drug Administration granted accelerated approval to Jemperli for adults with previously untreated stage II or stage III locally advanced rectal cancer whose tumors are mismatch repair deficient or microsatellite instability-high. Eligibility must be determined by an FDA-authorized test. Approximately 3 percent of locally advanced rectal cancers are mismatch repair deficient or microsatellite instability-high.

The FDA based its decision on interim results from AZUR-1, a phase 2 study evaluating Jemperli in 154 patients. Patients received up to nine doses given every 3 weeks over 6 months. After a median follow-up of more than 2 years, almost three-fourths of patients had not needed surgery, radiotherapy or chemotherapy.

An earlier study at Memorial Sloan Kettering Cancer Center evaluated Jemperli in 50 patients. None had detectable signs of cancer after treatment, and 93 percent maintained cancer-free status for at least 12 months. Of 27 patients followed for at least 3 years, only one had cancer return.

The most common side effects reported in at least 15 percent of patients were fatigue, rash, diarrhea and itching. Jemperli can cause the immune system to attack healthy organs and tissues. These reactions can be severe or fatal.

Acting FDA Commissioner Kyle Diamantas said the review was completed nearly 4 months ahead of schedule. The FDA granted Priority Review and Breakthrough Therapy designation. This marks the 11th approval under the Commissioner’s National Priority Voucher pilot program. The approval was granted to GlaxoSmithKline.