FDA News

FDA grants priority review to Azedra for rare neuroendocrine tumors

The FDA granted priority review to iobenguane I 131 for the treatment of patients with malignant, recurrent or unresectable pheochromocytoma and paraganglioma, according to the agent’s manufacturer.

There are no FDA-approved treatments for these rare neuroendocrine tumors.

Iobenguane I 131 (Azedra, Progenics Pharmaceuticals) is a substrate for norepinephrine reuptake transporter, which is highly expressed in neuroendocrine tumors. The FDA previously granted orphan drug and breakthrough therapy designations to the agent.

The FDA based the priority review designation on results of an open-label, multicenter phase 2b trial — conducted under a special protocol assessment agreement with the FDA — of patients with malignant, recurrent or unresectable pheochromocytoma and paraganglioma.

The study met its primary endpoint of 50% or greater reduction of all antihypertensive medication for at least 6 months. Results also showed associations with overall tumor response as measured by RECIST criteria.

The investigators found iobenguane I 131 to be safe and generally well tolerated.

“With no FDA-approved therapies for these rare tumors, Azedra has the potential to address the high unmet need of patients with malignant pheochromocytoma and paraganglioma,” Mark Baker, CEO of Progenics, said in a company-issued press release. “We are pleased that the FDA has accepted our [new drug application] with priority review and look forward to working with the agency during the review process. At the same time, we will continue to lay the groundwork for our commercial plan and prepare to launch quickly following a potential approval.”

The FDA is expected to make a decision on iobenguane I 131’s approval status by April 30.

The FDA granted priority review to iobenguane I 131 for the treatment of patients with malignant, recurrent or unresectable pheochromocytoma and paraganglioma, according to the agent’s manufacturer.

There are no FDA-approved treatments for these rare neuroendocrine tumors.

Iobenguane I 131 (Azedra, Progenics Pharmaceuticals) is a substrate for norepinephrine reuptake transporter, which is highly expressed in neuroendocrine tumors. The FDA previously granted orphan drug and breakthrough therapy designations to the agent.

The FDA based the priority review designation on results of an open-label, multicenter phase 2b trial — conducted under a special protocol assessment agreement with the FDA — of patients with malignant, recurrent or unresectable pheochromocytoma and paraganglioma.

The study met its primary endpoint of 50% or greater reduction of all antihypertensive medication for at least 6 months. Results also showed associations with overall tumor response as measured by RECIST criteria.

The investigators found iobenguane I 131 to be safe and generally well tolerated.

“With no FDA-approved therapies for these rare tumors, Azedra has the potential to address the high unmet need of patients with malignant pheochromocytoma and paraganglioma,” Mark Baker, CEO of Progenics, said in a company-issued press release. “We are pleased that the FDA has accepted our [new drug application] with priority review and look forward to working with the agency during the review process. At the same time, we will continue to lay the groundwork for our commercial plan and prepare to launch quickly following a potential approval.”

The FDA is expected to make a decision on iobenguane I 131’s approval status by April 30.