The Food and Drug Administration has approved the first radiopharmaceutical, lutetium Lu 177 dotatate (Lutathera), for the treatment of somatostatin receptor positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors in adults.

Approval is based on two studies, including the phase 3, NETTER-1, that compared lutetium Lu 177 dotatate plus octreotide to octreotide alone, and a subset of patients from an expanded access program in the Netherlands in patients with somatostatin receptor positive tumors, the FDA said in a statement .

The NETTER-1 study included patients who had inoperable midgut NETs progressing under standard dose octreotide treatment and overexpressing somatostatin receptors. The primary endpoint was met, showing a 79% reduction in risk of disease progression or death in the study arm compared to the control (hazard ratio, 0.21, 95% confidence interval, 0.13-0.32, P less than. 0001). There was a 48% reduction in the estimated risk of death with lutetium Lu 177 dotatate treatment compared to treatment with octreotide alone at a preplanned interim overall survival analysis (hazard ratio, 0.52, 95% confidence interval, 0.32-0.84).

In the expanded access study, complete or partial tumor shrinkage was reported in 16% of the patients in the subset of 360 patients with GEP-NETs.

Common side effects include lymphopenia, increased GGT, AST and/or ALT, vomiting, nausea, hyperglycemia and hypokalemia.

Serious side effects include myelosuppression, secondary myelodysplastic syndrome and leukemia, renal toxicity, hepatotoxicity, neuroendocrine hormonal crises, and infertility. Patients taking lutetium Lu 177 dotatate are exposed to radiation and exposure of other patients, medical personnel, and household members should be limited in accordance with radiation safety practices, the FDA said.