FDA Approves Lutetium Lu-177 Dotatate for Treatment of Gastroenteropancreatic Neuroendocrine Tumors


On January 26, the U.S. Food and Drug Administration (FDA) approved lutetium Lu-177 dotatate (Lutathera) for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs). This is the first time a radiopharmaceutical has been approved for the treatment of GEP-NETs. Lu-177 dotatate is indicated for adult patients with somatostatin receptor–positive GEP-NETs.

“GEP-NETs are a rare group of cancers with limited treatment options after initial therapy fails to keep the cancer from growing,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This approval provides another treatment choice for patients with these rare cancers. It also demonstrates how the FDA may consider data from therapies that are used in an expanded access program to support approval for a new treatment.”

Lu-177 dotatate is a radioactive drug that works by binding the somatostatin receptor, which may be present on certain tumors. After binding to the receptor, the drug enters the cell, allowing radiation to cause damage to the tumor cells.

Study Results

The approval of Lu-177 dotatate was supported by two studies. The first was a randomized clinical trial in 229 patients with a certain type of advanced somatostatin receptor–positive GEP-NET. Patients in the trial either received Lu-177 dotatate in combination with the drug octreotide or octreotide alone. The study measured progression-free survival. Progression-free survival was longer for patients taking Lu-177 dotatate with octreotide compared to patients who received octreotide alone.

The second study was based on data from 1,214 patients with somatostatin receptor–positive tumors, including GEP-NETS, who received Lu-177 dotatate at a single site in the Netherlands. Complete or partial tumor shrinkage was reported in 16% of a subset of 360 patients with GEP-NETs who were evaluated for response by the FDA. Patients initially enrolled in the study received Lu-177 dotatate as part of an expanded access program.

Adverse Events and Safety

Common side effects of Lu-177 dotatate include lymphopenia, high levels of enzymes in certain organs (increased GGT, AST, and/or ALT), vomiting, nausea, hyperglycemia, and hypokalemia.

Serious side effects of Lu-177 dotatate include myelosuppression, secondary myelodysplastic syndrome and leukemia, renal toxicity, hepatotoxicity, neuroendocrine hormonal crises, and infertility. Lu-177 dotatate can cause harm to a developing fetus; women should be advised of the potential risk to the fetus and to use effective contraception. Patients taking Lu-177 dotatate are exposed to radiation. Exposure of other patients, medical personnel, and household members should be limited in accordance with radiation safety practices.

The FDA granted the approval of Lu-177 dotatate to Advanced Accelerator Applications.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.