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Azedra: First treatment for rare adrenal tumors approved by FDA

The US Food and Drug Administration (FDA) has approved Azedra (iobenguane I 131), the first drug approved for treatment of rare unresectable adrenal gland tumors that spread beyond the original tumor site and need treating with a systemic anti-cancer therapy. 

Adrenal glands, situated above the kidneys, are responsible for producing several important hormones in the body, including stress hormones (epinephrines and norepinephrines), androgens, and aldosterone, which maintains the body’s salt and water levels and plays an important role in regulating blood pressure.  

Pheochromocytomas are tumors that form in the adrenal glands and these account for approximately nine in ten cases, according to the National Organization for Rare Disorders, whereas paragangliomas, which make up the remaining ten percent of cases, form outside the glands. 

They increase production of stress hormones, leading to hypertension and a variety of related symptoms, including headaches, sweating, a rapid heart rate, weight loss, nausea, vomiting, irritability, weakness, chest pain, and anxiety.

“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, 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.

Among the medications physicians may prescribe to treat hypertension secondary to pheochromocytoma are antihypertensives, such as Demser (metyrosine), and alpha-blocker antihypertensives, for example, Dibenzyline (phenoxybenzamine hydrochloride) or Minipress (prazosin).

In a clinical trial, the drug was shown to reduce tumor size in over one-fifth of trial participants. It also demonstrated Azedra’s efficacy in reducing the need for blood pressure medication in these patients. 

In addition to potentially reducing the size of tumors, the latest drug approved by the FDA also provides patients with access to an approved therapy shown to reduce the need for anti-hypertensives, Pazdur added. 

Fast Track, Breakthrough Therapy and Priority Review and Orphan Drug designations for Azedra were granted to Progenics Pharmaceuticals. 

What are the side effects of Azedra?

Among the most common severe side effects experienced by patients in the Azedra clinical trial were lymphopenia (low white blood cell count), neutropenia (a low count of a certain variety of white blood cell), thrombocytopenia (low blood platelet levels), fatigue, anemia, nausea, dizziness, hypertension, and vomiting. Some patients also experienced an increase reading in a laboratory test measuring blood clotting. 

Other warnings and precautions included a risk of lower blood cell levels, underactive thyroid, blood pressure elevations, renal failure, kidney injury, inflammation of lung tissues, and the dangers of exposure to radiation. The labeling warns that exposure should be minimized in pediatric patients and that family members should also minimize their exposure. 

Women prescribed Azedra are advised to take steps to avoid pregnancy due to the danger the drug will damage the unborn fetus.

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