FDA approved iobenguane I 131 (Azedra, Progenics Pharmaceuticals) injection for IV use to treat rare tumors of the adrenal gland.
Azedra is for adrenal tumors that cannot be surgically removed, have spread beyond the original tumor site, and require systemic anticancer therapy, in patients aged 12 years and older.
“Many patients with these ultra-rare cancers can be treated with surgery or local therapies, but there are no effective systemic treatments for patients who experience tumor-related symptoms such as high blood pressure,” said Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, in an FDA statement. “Patients will now have an approved therapy that has been shown to decrease the need for blood pressure medication and reduce tumor size in some patients.”
Azedra’s efficacy was shown in a single-arm, open-label, clinical trial in 68 patients. A quarter of the patients experienced a 50% or greater reduction of all antihypertensive medication for at least 6 months. Overall tumor response was achieved in 22% of the patients studied.
Because it is a radioactive therapeutic agent, Azedra includes a warning about radiation exposure to patients and family members, which should be minimized while the patient is receiving Azedra.
Myelodysplastic syndrome and acute leukemias, which are cancers of the blood and bone marrow, were observed in patients who received Azedra. FDA said the magnitude of this risk will continue to be studied.