Progenics Pharmaceuticals, for targeting on and treating cancer an oncology company creating innovative medicines and imaging analytical tools, reported that us food and drug administration (FDA) has accepted for review the new drug application (NDA) for Azedra in patients with malignant, recurrent or unresectable pheochromocytoma and paraganglioma, which are uncommon neuroendocrine tumors.
The FDA approved Progenics’ request under the Prescription Drug User Fee Act (PDUFA) for Priority Review has set an activity date of April 30, 2018. Mark Baker, a chief executive officer of Progenics said, “With no FDA- approved treatments for these uncommon tumors, Azedra can possibly address the high unmet need of patients with malignant pheochromocytoma and paraganglioma”.
“We are thankful that our NDA has accepted by the FDA with Priority Review, during the review process looking forward to working with the Agency. In the meantime, we will keep on laying the groundwork for our commercial plan and prepare to launch quickly following a potential approval.”
Under a Special Protocol Assessment (SPA) with the FDA, the NDA is supported by information from a pivotal phase 2b open-label, multicenter trial that was conducted.
The trial met the primary endpoint evaluating the proportion of pheochromocytoma and paraganglioma patients who achieved a half or greater reduction of all antihypertensive medication for no less than a half year, and demonstrated favorable outcomes from a key secondary endpoint evaluating the proportion of patients with overall tumor response as measured by Response Evaluation Criteria In Solid Tumors (RECIST).
Azedra was additionally appeared to be safe and generally well tolerated. Azedra (iobenguane I 131) being developed as a treatment for malignant, recurrent, or unresectable pheochromocytoma and paraganglioma, is a high- specific activity radiotherapeutic product candidate which are uncommon neuroendocrine tumors of neural crest origin.
Azedra is a substrate for norepinephrine reuptake transporter which is profoundly expressed on the cell surface of neuroendocrine tumors. In the US, Azedra has been granted Orphan Drug designation, Fast Track status, and Breakthrough Therapy designation.
Under an SPA agreement with the FDA, a phase 2b pivotal study has been completed in patients with malignant, recurrent, or unresectable pheochromocytoma and paraganglioma. There are currently no FDA- approved treatments for the treatment of this ultra-rare disease.