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. 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.
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.
Azedra is a radio-therapeutic compound 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 is also being evaluated 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 are currently limited. Malignant and recurrent pheochromocytomas and paragangliomas are often unresectable with very limited treatment options and no anti-tumor therapies are currently available.