The FDA has granted approval to iobenguane I 131 (Azedra, Progenics Pharmaceuticals, Inc.) for the treatment of iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL)—rare neuroendocrine tumors with a poor prognosis and limited treatment options—in adult and pediatric patients who require systemic anticancer therapy.
Approval was based on an open-label, single-arm, multicenter clinical trial which evaluated 68 patients (aged ≥12 years) with iobenguane scan-positive, unresectable, locally advanced or metastatic PPGL. Iobenguane I 131 produced an overall response rate of 22%, with 53% achieving a response duration of ≥6 months. Additionally, 25% of patients experienced a ≥50% reduction of antihypertensive medication for at least 6 months.
"Iobenguane is a true breakthrough in treating pheochromocytoma and paraganglioma, delivering an effective anticancer therapy to these tumors," said Daniel Pryma, MD, lead investigator and Chief, Division of Nuclear Medicine & Clinical Molecular Imaging at the Perelman School of Medicine at the University of Pennsylvania. "With this innovative, rationally designed treatment, we finally have a therapeutic option that helps address patients' needs."
For More Information
Clinicaltrials.gov (2018). A study evaluating ultratrace iobenguane I131 in patients with malignant relapsed/refractory pheochromocytoma/paraganglioma. NLM identifier: NCT00874614.
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