Azedra Proven FDA Approved Treatment for Adrenal Tumors
The US Food and Drug Administration has approved Azedra (iobenguane I 131) injection as the first FDA approved treatment adrenal tumors drug for adults and adolescents aged 12 and older with rare adrenal gland tumors known as pheochromocytoma or paraganglioma. The approval applies specifically to patients whose tumors cannot be surgically removed, have spread beyond the original tumor site, and require systemic anticancer therapy. According to the Mayo Clinic, pheochromocytomas are rare tumors that cause the adrenal glands to produce excess stress hormones, leading to dangerous elevations in blood pressure and a range of serious symptoms.
What Are Pheochromocytoma and Paraganglioma
Pheochromocytomas are rare tumors of the adrenal glands, located directly above the kidneys. These glands produce hormones including epinephrine and norepinephrine. When a pheochromocytoma develops, it increases production of these stress hormones, causing hypertension, headaches, rapid heart rate, sweating, nausea, vomiting, weight loss, chest pain, and anxiety.
When a tumor of the same type develops outside the adrenal gland, it is classified as a paraganglioma. Prior to the approval of this FDA approved treatment adrenal tumors therapy, no effective systemic treatment existed for patients whose disease had progressed beyond the reach of surgery or local therapies.
How Azedra Works
Azedra is a radioactive therapeutic agent administered by intravenous injection. As Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, noted, patients with these ultra rare cancers previously had no approved therapy capable of addressing tumor related symptoms such as high blood pressure at a systemic level. This FDA approved treatment adrenal tumors drug has now demonstrated it can decrease the need for blood pressure medication and reduce tumor size in a meaningful subset of patients.
Key Clinical Trial Results
The efficacy of Azedra was established in a single arm, open label clinical trial of 68 patients. The primary endpoint measured the proportion of patients who achieved a 50% or greater reduction in all antihypertensive medications lasting at least six months.
The trial met its primary endpoint. Seventeen of the 68 evaluable patients, or 25%, achieved a 50% or greater reduction in antihypertensive medication use for at least six months. Overall tumor response, measured by traditional imaging criteria, was achieved in 15 patients, representing 22% of those studied. These results established Azedra as a meaningful FDA approved treatment adrenal tumors option for a patient population with very limited alternatives.
Safety Profile and Warnings
The most common severe side effects in the clinical trial included low white blood cell counts, neutropenia, low platelet counts, fatigue, anemia, nausea, dizziness, hypertension, and vomiting.
Because Azedra is a radioactive agent, radiation exposure to patients and family members must be minimized during treatment. The risk of radiation exposure is higher in pediatric patients. Additional warnings include myelosuppression, underactive thyroid, blood pressure elevation, kidney injury, and lung tissue inflammation. Cases of myelodysplastic syndrome and acute leukemia were observed in patients who received Azedra, and the magnitude of this risk will continue to be studied. Women of childbearing age should use effective contraception following treatment, as Azedra can harm a developing fetus. Radiation exposure associated with this FDA approved treatment adrenal tumors drug may also cause infertility in both males and females.
Regulatory Designations
The FDA granted the Azedra application Fast Track, Breakthrough Therapy, Priority Review, and Orphan Drug designations. The Orphan Drug designation reflects the rarity of pheochromocytoma and paraganglioma and provides incentives to support development of therapies for rare diseases. Approval was granted to Progenics Pharmaceuticals, Inc.
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