The Food and Drug Administration (FDA) has approved Azedra (iobenguane I 131; Progenics) for the treatment of adult and pediatric patients ≥12 years old with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy. Azedra is the first FDA-approved treatment for this use. 

Azedra, a radioactive therapeutic agent, is similar in structure to the neurotransmitter norepinephrine (NE) and is subject to the same uptake and accumulation pathways as NE. Following IV administration, Azedra is taken up and accumulates within pheochromocytoma and paraganglioma cells, and radiation resulting from radioactive decay of I 131 causes cell death and tumor necrosis. The proprietary technology prevents unlabeled or “cold” iobenguane from being carried through to the final formulation, thus producing a drug with high specific activity. 

Azedra was evaluated in a single-arm, open-label trial (N=68) that measured the number of patients who experienced a ≥50% reduction of all antihypertensive drugs for ≥6 months. A secondary endpoint was overall tumor response, which was measured by traditional imaging criteria. The study achieved the primary endpoint as 25% of patients experienced a ≥50% reduction of all antihypertensive medications for ≥6 months. With regard to to the secondary endpoint, overall tumor response was seen in 22% of evaluable patients. 

Lymphopenia, neutropenia, thrombocytopenia, fatigue, anemia, increased INR, nausea, dizziness, hypertension, and vomiting were the most common adverse effects reported in clinical trials. 

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Azedra should be used by or under the control of physicians who are qualified by specific training and experience in the safe use and handling of radiopharmaceuticals, and whose experience and training have been approved by the appropriate governmental agency authorized to license the use of radiopharmaceuticals. It will be available as 30mL single-dose vials containing 555MBq/mL of I-131 and 0.006mg/mL of iobenguane. 

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