New Treatment for Pheochromocytoma and Paraganglioma Approved by the FDA

According to a story from, the company Progenic Pharmaceuticals recently reported the the U.S. Food and Drug Administration has recently approved the company’s product iobenguane I 131 (AZEDRA). This radiotherapy injection is indicated for the treatment of pheochromocytoma and paraganglioma tumors for patients 12 years or older. The patients must present with either metastatic or locally advanced disease that is not resectable and is iobenguane scan positive. This historic approval makes AZEDRA the only therapy that has gained approval for addressing this specific medical need.

About The Neuroendocrine Tumors (Pheochromocytoma and Paraganglioma)

Pheochromocytoma and paraganglioma are two rather similar and rare neuroendocrine tumors. Pheochromocytomas and paragangliomas originate from chromaffin cells and glomus cells, respectively. In about a quarter of cases, these tumors are heritable and are linked to several different genetic mutations. The rest of cases appear spontaneously.

Pheochromocytoma occurs in the medulla of the adrenal glands. Symptoms of the tumor and indicative of hyperactivity of the sympathetic nervous system. They include high blood pressure and heartrate, palpitations, anxiety/panic attacks, flank pain, skin sensations, amyloid deposits, elevate blood glucose, weight loss, heavy sweating, and headaches. A potential complication is arterial hypertension, which can result in a medical emergency. Symptoms for paraganglioma are similar, but they do not always appear. Treatment typically involves surgery or radiation therapy. If paraganglioma metastasizes, the five year survival rate is 45 percent.

A Better Chance For Patients

It is generally quite infrequent that either of these neuroendocrine tumors become malignant and develop into cancer, but when they do, the outcomes for patients have historically not been very good, with relatively poor rates of long term survival. There is also no test that can determine whether a paraganglioma is benign or malignant, which is a disadvantage for patients in comparison to other types of tumors. This means that patients are more or less forced to monitor their tumors long term. When these neuroendocrine tumors do spread, the frontline treatment of surgical resection becomes ineffective. This is where AZEDRA comes in. Now patients with advanced disease will have a much greater chance against these tumors.

Side Effects

While the treatment can help this group of patients, it is worth mentioning that AZEDRA use comes with the potential for serious side effects, such as risks associated with radiation exposure, low blood cells counts, high blood pressure, and thyroid and kidney problems.

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