The FDA has approved iobenguane I-131 (Azedra) for adult and pediatric patients aged â‰¥12 years with iobenguane scanâ€“positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy.
The FDA has approved iobenguane I-131 (Azedra) for adult and pediatric patients aged ≥12 years with iobenguane scan—positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL) who require systemic anticancer therapy.
The approval was based on findings from a phase IIb clinical trial that was conducted under the FDA's special protocol assessment. In the trial, the radiopharmaceutical elicited a ≥50% reduction in antihypertensive medication use for ≥6 months, which was the primary endpoint of the trial, for 25% of patients with PPGL (95% CI, 16%-37%). In those receiving at least 2 therapeutic doses, the primary endpoint was achieved for 32% of patients (95% CI, 21%-46%).
“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,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a statement.
“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,” added Pazdur.
Iobenguane I-131 was previously granted a breakthrough therapy designation for the treatment of PPGL, along with orphan drug and fast track designations.
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