FDA approves radiotherapeutic for treatment of ultra-rare adrenal gland tumours

An intravenous injection — Azedra (iobenguane I 131) — for the treatment of adults and adolescents age 12 and older with rare tumours of the adrenal gland that are unresectable, has been approved by the US Food and Drug Administration (FDA).

“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 tumour-related symptoms such as high blood pressure,” said Dr Richard Pazdur, 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. “Patients will now have an approved therapy that has been shown to decrease the need for blood pressure medication and reduce tumour size in some patients.”

Azedra (from Progenics Pharmaceuticals) is a radiotherapeutic indicated for the treatment of patients (adults and those 12 years and older) with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma where system anticancer therapy is required.

Pheochromocytoma and paragaglioma are neuroendocrine cancers that occur in cells of the adrenal glands. They frequently secrete high levels of hormones that can lead to life-threatening high blood pressure, heart failure and stroke.

“Azedra is a true breakthrough in treating pheochromocytoma and paraganglioma delivering an effective anticancer therapy to these tumours,” stated Dr Daniel Pryma, associate professor of Radiology & Radiation Oncology and chief, Division of Nuclear Medicine & Clinical Molecular Imaging at the Perelman School of Medicine at the University of Pennsylvania. “With this innovative, rationally designed treatment, we finally have a therapeutic option that helps address patients’ needs.”

“As the first FDA approved therapy for unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy, Azedra provides a new treatment option for physicians and their patients,” added Mark Baker, chief executive officer of Progenics. “Azedra has been shown to decrease the need for blood pressure medication and reduce tumour size in some patients. We are extremely grateful to the patients, their families and the investigators who participated in Azedra’s clinical development program. We also thank those who have contributed to the development of Azedra over many years.”

Emily Collins, president of the Pheo Para Alliance commented: “The FDA's approval of Azedra is welcome news to patients with pheochromocytoma and paraganglioma, who have an extremely limited number of treatment options available to them. The drug's Fast-Track status and Breakthrough Therapy designation by the FDA underscores the dire need for the development and expeditious review of diagnostic and therapeutic agents for pheo/para that, generally, don’t get adequate prioritisation despite the growing prevalence of these and other NET cancers globally.”

This approval by the FDA was based on the results of a Phase II open-label, multicentre trial that was conducted under a special protocol assessment (SPA) with the FDA.