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The American Food & Drug Administration (FDA) just approved Lutetium (Lu 177) dotatate (Lutathera) for the treatment of GEP-NETs. This is the first time a radioactive drug, or radiopharmaceutical, has been approved for the treatment of GEP-NETs by the FDA. Lutetium (Lu 177) dotatate (Lutathera) is indicated for adult patients with somatostatin receptor-positive GEP-NETs.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.
GEP-NETs can be present in the pancreas and in different parts of the gastrointestinal tract such as the stomach, intestines, colon and rectum. It is estimated that approximately one out of 27,000 people are diagnosed with GEP-NETs per year. Lutetium (Lu 177) dotatate (Lutathera) is a radioactive drug that works by binding to 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.
The approval of Lutetium (Lu 177) dotatate (Lutathera) was supported by two clinical studies. The first was a randomized clinical trial in 229 patients with a certain type of advanced somatostatin receptor-positive GEP-NET. Patients in this trial either received Lutetium (Lu 177) dotatate (Lutathera) in combination with the drug octreotide or octreotide alone. The study measured the length of time the tumors did not grow after treatment (so-called progression-free survival). Progression-free survival was longer for patients taking Lutetium (Lu 177) dotatate (Lutathera) with octreotide compared to patients who received octreotide alone. This means the risk of tumor growth or patient death was lower for patients who received Lutathera with octreotide compared to that of patients who received only octreotide.
The second study was based on data from 1,214 patients with somatostatin receptor-positive tumors, including GEP-NETS, who received Lutetium (Lu 177) dotatate (Lutathera) at a single site in the Netherlands. Complete or partial tumor shrinkage was reported in 16 percent of a subset of 360 patients with GEP-NETs who were evaluated for response by the FDA. Patients initially enrolled in the study received Lutetium (Lu 177) dotatate (Lutathera) as part of an expanded access program. Expanded access is a way for patients with serious or immediately life-threatening diseases or conditions who lack therapeutic alternatives to gain access to investigational drugs for treatment use.
As is the case with many therapies, Lutetium (Lu 177) dotatate comes with a fair and serious array of serious adverse effects, some of which may be life threathening. Common side effects of Lutetium (Lu 177) dotatate (Lutathera) include low levels of white blood cells (lymphopenia), high levels of enzymes in certain organs (increased GGT, AST and/or ALT), vomiting, nausea, high levels of blood sugar (hyperglycemia) and low levels of potassium in the blood (hypokalemia).
Serious and possibly liefe threatening adverse effects of Lutetium (Lu 177) dotatate (Lutathera) include low levels of blood cells (myelosuppression), development of certain blood or bone marrow cancers (secondary myelodysplastic syndrome and leukemia), kidney damage (renal toxicity), liver damage (hepatotoxicity), abnormal levels of hormones in the body (neuroendocrine hormonal crises) and infertility. Lutetium (Lu 177) dotatate (Lutathera) 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 Lutetium (Lu 177) dotatate (Lutathera) are exposed to radiation. Exposure of other patients, medical personnel, and household members should be limited in accordance with radiation safety practices.
Note that Lutetium (Lu 177) dotatate (Lutathera) had already been approved by the European Medicines Agency (EMA) in September 2017.