On January 26, 2018, the FDA approved lutetium Lu 177 dotatate (Lutathera; Advanced Accelerator Applications), a radioactive drug, for the treatment of adults with somatostatin receptor–positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Lutetium Lu 177 dotatate was approved under the FDA’s priority review process, and was designated as an orphan drug.
This is the first approval of a radioactive drug for the treatment of patients with GEP-NETs. This radioactive drug works by binding to a somastatin receptor on the tumor, allowing the radiation to destroy the cancerous cells.
“GEP-NETs are a rare group of cancers with limited treatment options after initial therapy fails to keep the cancer from growing. 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,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence.
This approval was based on the results of 2 clinical trials. The first was a study of 229 patients with a particular type of advanced somatostatin receptor–positive GEP-NET who were randomized to lutetium Lu 177 dotatate plus octreotide or octreotide monotherapy. The end point was progression-free survival, which was longer in patients who received lutetium Lu 177 dotatate plus octreotide than in those who received octreotide monotherapy, translating to a lower risk for death or tumor growth in those who received the combination versus monotherapy.
The second study included 1214 patients with somatostatin receptor–positive tumors, including GEP-NETs. Among a subgroup of 360 patients with GEP-NETs who received lutetium Lu 177 dotatate and were assessed by the FDA, 16% had complete or partial response.
The common side effects associated with lutetium Lu 177 dotatate include lymphopenia, high enzyme levels in certain organs, vomiting, nausea, hyperglycemia, and hypokalemia. Serious side effects include myelosuppression, secondary myelodysplastic syndrome, leukemia, renal toxicity, hepatotoxicity, neuroendocrine hormonal crises, and infertility.
Patients receiving lutetium Lu 177 dotatate are exposed to radiation, which may cause harm to a developing fetus and to other people.