Therapy for rare cancers receives FDA approval

The U.S. Food and Drug Administration (FDA) has approved the first ever non-surgical treatment for the rare neuroendocrine cancers pheochromocytoma and paraganglioma. The approval was based on a multi-center trial led by researchers in the Abramson Cancer Center, and was granted to Progenics Pharmaceuticals for AZEDRA (iobenguane I131).

Daniel A. Pryma, associate professor at the Perelman School of Medicine

“This is a true breakthrough. Until today, there were no anti-tumor therapies available for patients with these tumors who were not candidates for surgery,” said the trial’s principal investigator Daniel A. Pryma, an associate professor of radiology and radiation oncology, chief of nuclear medicine and clinical molecular imaging at the Perelman School of Medicine, and a member of the Abramson Cancer Center.

AZEDRA is a targeted, high-specific-activity radiotherapeutic, indicated for the treatment of adult and pediatric patients (12 years and older) with a positive iobenguane scan, and unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy. AZEDRA is the first and only approved therapy for this indication.

Read more at Penn Medicine News.