FDA approves first treatment for rare adrenal tumors

FDA approved on Monday iobenguane I 131 (Azedra—Progenics Pharmaceuticals) injection for the treatment of rare tumors of the adrenal gland (pheochromocytoma or paraganglioma) that are unresectable, have spread beyond the original tumor site, and require systemic anticancer therapy.

FDA approved on Monday iobenguane I 131 (Azedra—Progenics Pharmaceuticals) injection for the treatment of rare tumors of the adrenal gland (pheochromocytoma or paraganglioma) that are unresectable, have spread beyond the original tumor site, and require systemic anticancer therapy. The drug was approved for use in adults and adolescents 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. "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." Commonly reported severe adverse effects reported by patients taking iobenguane I 131 in clinical trials included lymphopenia, neutropenia, thrombocytopenia, fatigue, anemia, increased international normalized ratio, nausea, dizziness, hypertension, and vomiting. The drug, a radioactive therapeutic agent, includes a warning about radiation exposure to patients and family members. There is also a risk of myelosuppression, underactive thyroid, elevations in blood pressure, renal failure or kidney injury, and pneumonitis. Additionally, myelodysplastic syndrome and acute leukemias were observed in patients who took iobenguane I 131, and the magnitude of this risk will continue to be studied.