FDA approves first-in-class treatment for rare adrenal tumors

The US FDA has approved Adzera® (iobenguane I 131) for intravenous use for the treatment of patients age 12 or older with rare tumors of the adrenal gland, pheochromocytoma and paraganglioma. This is the first approved drug to treat tumors of this kind that are unresectable, have spread beyond the primary location and require systemic anticancer therapy.

“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,” commented Richard Pazdur, 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.

“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.”

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Azedra’s efficacy was demonstrated in a single-arm, open-label clinical trial that enrolled 68 patients. The primary endpoint of the study was patients who experienced at least a 50% reduction in antihypertensive medications lasting at least six months, backed up by the secondary endpoint of overall tumor response, which was measured by traditional imaging. The study met the primary endpoint in 25% patients, and the secondary endpoint in 22%.

Iobenguane I 131 is a radioactive therapeutic agent and thus comes with a warning detailing radiation exposure to patients and family members; exposure risk is greater in younger patients. The FDA granted this application Fast Track, Breakthrough Therapy and Priority Review designations, as well as Orphan Drug designation to encourage the development of drugs for these rare tumors.

Source: www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm615155.htm