Therapy for Pheo and Para Neuroendocrine Tumors Receives FDA Approval Following Trials at the University of Pennsylvania

On July 30, Progenics Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) has approved the New Drug Application (NDA) for AZEDRA (iobenguane I 131) 555 MBq/mL injection for intravenous use. AZEDRA, a radiotherapeutic, is indicated for the treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy.  AZEDRA is the first and only approved therapy for this indication.

 NANETS Member Daniel M. Pryma, MD, the clinical trial's lead investigator, said, "AZEDRA is a true breakthrough in treating pheochromocytoma and paraganglioma delivering an effective banticancer therapy to these tumors. With this innovative, rationally designed treatment, we finally have a therapeutic option that helps address patients’ needs." 

Pheochromocytoma and paraganglioma are neuroendocrine cancers that arise from cells in and around the adrenal glands. Pheochromocytoma and paraganglioma tumors frequently secrete high levels of hormones that can lead to life-threatening high blood pressure, heart failure, and stroke in these patients.  Metastatic pheochromocytoma and paraganglioma may result in unresectable disease with a poor prognosis, including a five-year survival rate as low as 12%1. Before AZEDRA’s approval, there were no FDA-approved anti-tumor therapeutics for these cancers.​

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A Single-Institution Retrospective Analysis of Metachronous and Synchronous Metastatic Bronchial Neuroendocrine Tumors
In one of the largest, single-center, series of metastatic broncho-pulmonary neuroendocrine tumors (bpNETs), researchers studied disease, patient, and treatment characteristics by retrospectively analyzing cases of bpNETs and distant metastases (metachronous or synchronous), evaluated at the European Institute of Oncology between 1997 and 2014. Heterogeneity was revealed in the pattern of recurrences and the follow-up exams among patients with metachronous metastases. Most common sites of relapse were liver (67%), lymph node (25%), bone (22%) and lung (16%), and the most common tool used to detect first recurrence was computed tomography. A significant correlation of age, bone metastases, liver metastases and Ki-67 as a continuous variable, with prognosis was indicated in the multivariate analysis.

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