Progenics Pharmaceuticals, Inc., announced the achievement of an important milestone that moves their orphan drug AZEDRA® (iobenguane I 131) Injection closer to requesting FDA-approval. AZEDRA is a targeted radiotherapeutic agent being evaluated as an imaging agent at low doses and a cancer-killing therapy at high doses for the treatment of malignant and/or recurrent pheochromocytoma/paraganglioma. Results from a phase 2b trial showed that treatment with AZEDRA resulted in significant reductions in the use of antihypertensive medications needed to control blood pressure. The announcement also referenced favorable data on overall tumor response, suggesting that AZEDRA may provide clinically meaningful impacts on the tumor. In addition, AZEDRA was reported to be generally well-tolerated.
As of today, there are no FDA-approved therapies for the treatment of malignant pheochromocytoma/paraganglioma. If approved, AZEDRA could be a promising step in improving the lives of patients with this rare disease. Mark Baker, CEO of Progenics, is anticipating the New Drug Application (or “NDA”) will be submitted to the FDA by mid-year and hopes that this drug will be offered expedited review, since it has been granted “breakthrough,”
“fast track,” and “orphan drug” status by FDA. The clinical trial’s lead investigator, 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, said, “With the lack of approved treatment options many patients are treated with the ‘kitchen sink’ approach [and] given a variety of toxic and ineffective treatments.” AZEDRA represents a treatment option that could help deliver lethal levels of radioactivity to the tumor and presents a targeted treatment therapy to our patient base.
We will keep you posted on any details we hear regarding the trial and the anticipated approval, which we hope will be a significant step in the right direction for our rare disease community.
AZEDRA® is a registered trademark of Molecular Insight Pharmaceuticals, Inc., a wholly owned subsidiary of Progenics Pharmaceuticals, Inc.