Dive Brief:
- The Food and Drug Administration on Monday OK'd a radiotherapeutic for a rare type of unresectable adrenal gland tumors, sending shares in the drug's maker Progenics Pharmaceuticals temporarily higher.
- Approval of Azedra was based on a pivotal Phase 2 study carried out under a special protocol assessment, in which a quarter of studied patients saw a 50% or greater reduction in their hypertensive medication — the primary endpoint of the study. Secondary endpoint results were "favorable," and tumor responses were seen in 22% of patients.
- Pheochromocytoma and paraganglioma, the two adrenal tumors tested, can cause life-threatening high blood pressure and subsequent heart failure and stroke. Between two million and eight million people are diagnosed globally each year, with 10% of cases seen in children.
Dive Insight:
Azedra (iobenguane I 131) is the first and only FDA-approved treatment for these rare neuroendocrine cancers. To speed the progress of getting the drug to market, regulators granted Azedra Priority Review as well as Orphan Drug, Fast Track and Breakthrough Therapy designations.
"Many patients with these ultra-rare cancers can be treated with surgery or local therapies, but there are no effective systemic treatments for patients who experience tumor-related symptoms such as high blood pressure," said Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.
Azedra's approval, however, was delayed from previous expectations. In March 2018, the FDA pushed the review date for the drug back three months to July 30, 2018, due to a need for extra time to assess additional chemistry, manufacturing, and controls information.
Treatment for these adrenal tumors include radiotherapy and chemotherapy, as well as procedures like surgery, ablation and embolization aimed at removing or reducing the tumors. There are few drugs in the global pipeline that specifically target pheochromocytoma and paraganglioma, so while it's a rare disorder and the market is small, Progenics could carve out its own niche.
Though investors appeared initially optimistic on news of the approval, sentiment seemed to shift Tuesday, when shares lost nearly 7% of their value in morning trading.