On July 28, 2015 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX), an oncology company developing innovative ways to target and treat cancer, reported that the U.S. Food and Drug Administration (FDA) has designated Azedra as a Breakthrough Therapy for the treatment of patients with iobenguane-avid metastatic or recurrent pheochromocytoma and paraganglioma (Press release, Progenics Pharmaceuticals, JUL 28, 2015, View Source [SID:1234506726]). Schedule your 30 min Free 1stOncology Demo! Azedra is currently being evaluated in a pivotal Phase 2b trial, which is being conducted under a Special Protocol Assessment Agreement (SPA), and has received Orphan Drug and Fast Track designations from the FDA.
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The Breakthrough Therapy designation pathway was created by the FDA to expedite the development and review for a drug that treats a serious or life-threatening condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies.
"This Breakthrough Therapy designation for Azedra reflects the urgent need for new options for patients suffering from pheochromocytoma and paraganglioma," stated Mark Baker, CEO of Progenics. "Azedra has the potential to be the first approved therapy for patients with these rare and devastating tumors, and this designation will allow for a close collaboration between Progenics and the FDA as we complete our ongoing pivotal Phase 2b trial and, assuming a positive trial outcome, advance Azedra through the regulatory review process."
About AZEDRA
Azedra is a radio-therapeutic with FDA Fast Track status currently being studied in a phase 2 registrational trial under a SPA with the FDA using a surrogate marker as registrational endpoint. In addition to potentially treating pheochromocytoma and paraganglioma, Azedra may also have utility in treating neuroblastoma and other neuroendocrine tumors. Clinical trials for Azedra have not been completed and regulatory approvals for Azedra remain subject to the successful completion of all required studies.
About Pheochromocytoma and Paraganglioma
Pheochromocytomas and paragangliomas are rare neuroendocrine tumors that arise from cells of the sympathetic nervous system. When pheochromocytomas are located outside the adrenal glands, they are called paragangliomas. Standard treatment options for these tumors include surgery, palliative therapy and symptom management. Malignant and recurrent pheochromocytomas and paragangliomas may result in unresectable disease with a poor prognosis, representing a significant management challenge with very limited treatment options and no approved anti-tumor therapies.