Rare Daily Staff

The U.S. Food and Drug Administration approved Progenics Pharmaceuticals’ Azedra, a radiotherapeutic for the treatment of adult and pediatric patients 12 years and older with rare neuroendocrine cancers, the first and only approved therapy for the indication.

Azedra is approved to treat pheochromocytoma and paraganglioma, neuroendocrine cancers that arise from cells in and around the adrenal glands. Pheochromocytoma and paraganglioma tumors frequently secrete high levels of hormones that can lead to life-threatening high blood pressure, heart failure, and stroke in these patients. Metastatic pheochromocytoma and paraganglioma may result in unresectable disease with a poor prognosis, including a five-year survival rate as low as 12 percent.

“Azedra is a true breakthrough in treating pheochromocytoma and paraganglioma delivering an effective anticancer therapy to these tumors,” said Daniel Pryma, associate professor of radiology and radiation oncology and chief, division of nuclear medicine and Clinical Molecular Imaging at the Perelman School of Medicine at the University of Pennsylvania, the trial’s lead investigator. “With this innovative, rationally designed treatment, we finally have a therapeutic option that helps address patients’ needs.”

The FDA’s approval of Azedra was based on data from a pivotal phase 2 open-label, multi-center trial that was conducted under a Special Protocol Assessment with the FDA. The results showed that 17 of the 68 evaluable patients (25 percent) experienced a 50 percent or greater reduction of all antihypertensive medication for at least 6 months, achieving the primary endpoint specified in the SPA.

The study also showed favorable results from a key secondary endpoint evaluating the proportion of patients with overall tumor response as measured by Response Evaluation Criteria In Solid Tumors (RECIST). Overall tumor response was achieved in 15 of the patients studied (22 percent). Of these 15 patients, 53 percent experienced durable tumor responses lasting six months or longer.

Azedra can cause serious side effects including risk from radiation exposure, bone marrow problems and other cancers, thyroid problems, elevations in blood pressure, kidney problems, respiratory problems. It carries a pregnancy warning and can cause fertility problems.

July 31, 2018
Photo: Daniel Pryma, associate professor of radiology and radiation oncology and chief, division of nuclear medicine and Clinical Molecular Imaging at the Perelman School of Medicine

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