Progenics Pharmaceuticals, a biotech company based in Tarrytown, NY, announced that the U.S. FDA has granted its Azedra™ (Ultratrace® iobenguane I 131) Breakthrough Therapy Designation for the treatment of patients with iobenguane-avid metastatic or recurrent pheochromocytoma and paraganglioma.  Pheochromocytoma is a rare endocrine tumor originating in the adrenal glands; while paraganglioma is a closely related tumor that originates from the sympathetic or parasympathetic nervous system outside the adrenal gland.   Surgical resection is the most common treatment of solitary pheochromocytomas and paragangliomas.  However, once these tumors progress that result in unresectable disease, the treatment options are very limited and the prognosis is poor.  Azedra is a radio-therapeutic consisting of a small molecule that specifically targets neuroendocrine tumors (such as pheochromocytoma and paraganglioma) and is also labeled with radioisotope serving both as an imaging agent and a therapeutic.  Doctors can first image with this radiopharmaceutical to determine a therapeutic dose that is safe to normal organs and effective in destroying cancer cells enabling the delivery of an individualized therapeutic dose optimal for each patient.  Azedra is currently under Phase II study evaluating its efficacy and safety in patients with malignant relapsed/refractory pheochromocytoma/paraganglioma.

 

References:

  1. http://ir.progenics.com/releasedetail.cfm?ReleaseID=924136
  2. https://clinicaltrials.gov/ct2/show/NCT00874614?term=Azedra&rank=1