FDA News

FDA grants breakthrough therapy designation to Azedra for pheochromocytoma, paraganglioma

The FDA granted breakthrough therapy designation to Ultratrace iobenguane I-131 for the treatment of patients with iobenguane-avid metastatic or recurrent pheochromocytoma and paraganglioma, according to a press release from the drug’s manufacturer.

Ultratrace iobenguane I-131 (Azedra, Progenics Pharma), a late-state radiotherapeutic drug candidate, is being evaluated in a phase 2b trial of patients with malignant pheochromocytoma or paraganglioma. Pheochromocytomas — rare tumors usually found within one or both adrenal glands — are referred to as paragangliomas when they arise in other areas of sympathetic nerve cells.

Ultratrace iobenguane I-131 — a very high specific activity form of iobenguane I-131 — is produced using Progenics’ Ultratrace platform, a technology designed to prevent unlabeled iobenguane from being carried through the manufacturing process to the final formulation, according to information listed on Progenics’ website. Ultratrace iobenguane I-131 is designed for use as an imaging agent in order to determine a therapeutic dose that is safe for normal organs, as well as for therapy.

The FDA previously granted orphan drug and fast track designations to Ultratrace iobenguane I-131.

"This breakthrough therapy designation for Azedra reflects the urgent need for new options for patients suffering from pheochromocytoma and paraganglioma," Mark R. Baker, CEO of Progenics Pharma, said in a press release. "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."

The FDA granted breakthrough therapy designation to Ultratrace iobenguane I-131 for the treatment of patients with iobenguane-avid metastatic or recurrent pheochromocytoma and paraganglioma, according to a press release from the drug’s manufacturer.

Ultratrace iobenguane I-131 (Azedra, Progenics Pharma), a late-state radiotherapeutic drug candidate, is being evaluated in a phase 2b trial of patients with malignant pheochromocytoma or paraganglioma. Pheochromocytomas — rare tumors usually found within one or both adrenal glands — are referred to as paragangliomas when they arise in other areas of sympathetic nerve cells.

Ultratrace iobenguane I-131 — a very high specific activity form of iobenguane I-131 — is produced using Progenics’ Ultratrace platform, a technology designed to prevent unlabeled iobenguane from being carried through the manufacturing process to the final formulation, according to information listed on Progenics’ website. Ultratrace iobenguane I-131 is designed for use as an imaging agent in order to determine a therapeutic dose that is safe for normal organs, as well as for therapy.

The FDA previously granted orphan drug and fast track designations to Ultratrace iobenguane I-131.

"This breakthrough therapy designation for Azedra reflects the urgent need for new options for patients suffering from pheochromocytoma and paraganglioma," Mark R. Baker, CEO of Progenics Pharma, said in a press release. "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."