FDA Accepts New Drug Application for Treatment of Rare Neuroendocrine Tumors
The radiotherapeutic product is a substrate for the norepinephrine reuptake transporter that is highly expressed on the cell surface of neuroendocrine tumors.
Progenics announced that the Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for Azedra (iobenguane I 131) for the treatment of malignant, recurrent and/or unresectable pheochromocytoma and paraganglioma.
Azedra, a high-specific-activity radiotherapeutic product, is a substrate for the norepinephrine reuptake transporter that is highly expressed on the cell surface of neuroendocrine tumors. The proprietary Ultratrace platform prevents unlabeled or "cold" iobenguane from being carried through the manufacturing process to the final formulation, resulting in a product with very high specific activity.
It has already been granted Orphan Drug designation, Fast Track status, and Breakthrough Therapy Designation by the FDA.
The NDA, which was accepted under Priority Review, included data from a Phase 2b open-label, multi-center trial that was conducted under a Special Protocol Assessment (SPA) with the FDA. The primary endpoint was met in evaluating patients with pheochromocytoma and paraganglioma who achieved a ≥50% reduction of all antihypertensive drugs for ≥6 months.
In addition, there was a favorable proportion of patients showing overall tumor response as measured by Response Evaluation Criteria in Solid Tumors (RECIST), which was a major secondary endpoint.
Regarding safety, the use of Azedra was generally well tolerated among study patients. A Prescription Drug User Fee Act (PDUFA) date has been set for April 30, 2018.
Progenics Pharmaceuticals announces FDA acceptance of New Drug Application for Azedra® (iobenguane I 131) in pheochromocytoma and paraganglioma [press release]. New York: Progenics Pharmaceuticals; December 29, 2017. Accessed January 4, 2018.