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July 30, 2018

On July 30, 2018, the Food and Drug Administration approved iobenguane ∣ 131 (AZEDRA, Progenics Pharmaceuticals, Inc.) for adult and pediatric patients (12 years and older) with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL) who require systemic anticancer therapy.

Approval was based on Study IB12B (NCT00874614), an open-label, single-arm, multicenter clinical trial in patients 12 years and older with iobenguane scan-positive, unresectable, locally advanced or metastatic PPGL. Of the 68 evaluable patients, 17 (25%; 95% CI: 16%, 37%) experienced a 50% or greater reduction of all antihypertensive medication for at least six months. Overall tumor response (RECIST 1.0) occurred in 15 patients (22%; 95% CI: 14%, 33%), with 53% achieving a response duration of at least 6 months.

The most common grade 3-4 adverse reactions (≥10%) were lymphopenia, neutropenia, thrombocytopenia, fatigue, anemia, increased international normalized ratio, nausea, dizziness, hypertension, and vomiting. In the pooled safety population, 6.8% of patients who received a therapeutic dose of iobenguane ∣ 131 developed myelodysplastic syndrome or acute leukemia.

Iobenguane ∣ 131 is administered in an initial dosimetric dose, followed by two therapeutic doses that are adjusted based on dosimetry. The recommended therapeutic dose is 18,500 MBq (500 mCi) for patients weighing more than 62.5 kg and 296 MBq/kg (8 mCi/kg) for patients 62.5 kg or less.

View full prescribing Information for Azedra.

FDA granted this application priority review, orphan product, fast track status, and breakthrough therapy designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System by completing a form online at, by faxing (1-800-FDA-0178) or mailing the postage-paid address form provided online, or by telephone (1-800-FDA-1088).