Treatment for Pheochromocytoma; Paraganglioma
Progenics Pharmaceuticals Completes Submission of NDA for Azedra (iobenguane I 131) in Pheochromocytoma and Paraganglioma
NEW YORK, Nov. 02, 2017 (GLOBE NEWSWIRE) — Progenics Pharmaceuticals, Inc. (NASDAQ:PGNX), an oncology company developing innovative medicines and imaging analytical tools for targeting and treating cancer, announced today that it has completed the rolling submission of its New Drug Application (NDA) for Azedra in patients with malignant, recurrent and/or unresectable pheochromocytoma and paraganglioma, which are rare neuroendocrine tumors.
Azedra has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA. There are currently no approved therapeutics in the U.S. for the treatment of malignant, recurrent, and/or unresectable pheochromocytoma or paraganglioma. The NDA remains subject to FDA regulatory review and approval.
“The completion of our NDA submission marks a significant milestone for Progenics and our Azedra development program,” said Mark Baker, Chief Executive Officer of Progenics. “If approved, Azedra has the potential to address a significant unmet need in pheochromocytoma and paraganglioma, an ultra-orphan indication.”
The NDA is supported by data from a pivotal Phase 2b open-label, multi-center trial that was conducted under a Special Protocol Assessment (SPA) with the FDA. The trial met the primary endpoint evaluating the proportion of pheochromocytoma and paraganglioma patients who achieved a 50% or greater reduction of all antihypertensive medication for at least six months, and showed favorable results from a key secondary endpoint evaluating the proportion of patients with overall tumor response as measured by Response Evaluation Criteria In Solid Tumors (RECIST) criteria. Azedra was also shown to be safe and generally well tolerated.
Azedra (iobenguane I 131), a radiotherapeutic product candidate in development as a treatment for malignant and/or recurrent pheochromocytoma and paraganglioma, rare tumors found in the adrenal glands and outside of the adrenal glands, respectively. Azedra has been granted Breakthrough Therapy and Orphan Drug designations, as well as Fast Track status in the U.S. Under a SPA agreement with the U.S. Food and Drug Administration (FDA), a Phase 2 pivotal study has been completed in patients with malignant, recurrent, and/or unresectable pheochromocytoma and paraganglioma. There are currently no FDA-approved therapies for the treatment of these ultra-orphan diseases.
About Pheochromocytoma and Paraganglioma
Pheochromocytoma and paraganglioma are rare neuroendocrine tumors that arise from cells of the autonomic 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. Pheochromocytoma and paraganglioma tumors frequently secrete high levels of hormones that can lead to life-threatening hypertension, heart failure, and stroke in these patients. Malignant and recurrent pheochromocytoma and paraganglioma 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.
Progenics develops innovative medicines and other technologies to target and treat cancer. Progenics’ pipeline includes: 1) therapeutic agents designed to precisely target cancer (Azedra® and 1095), 2) PSMA-targeted imaging agents for prostate cancer (1404 and PyL™), and 3) imaging analysis tools. Progenics’ first commercial product, RELISTOR® (methylnaltrexone bromide) for opioid-induced constipation, is partnered with Valeant Pharmaceuticals International, Inc.
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Source: Progenics Pharmaceuticals Inc.
Posted: November 2017
Azedra (iobenguane I 131) FDA Approval History