FDA approves first drug to treat rare adrenal cancer
Azedra has received FDA approval to become the first and only treatment for a type of rare adrenal cancer.
Progenics Pharmaceuticals shares soared after its drug Azedra, a new version of the radiopharmaceutical injectable drug iobenguane I-131, was given the green light by the US Food and Drug Administration (FDA).
The treatment is for patients aged 12 and older who have tumours of the adrenal gland that are unresectable – which means they cannot be surgically removed – and that are metastatic.
These uncommon neuroendocrine tumours of the adrenal glands are known as pheochromocytomas. They increase the production of epinephrines and norepinephrines, leading to hypertension and symptoms such as headaches, irritability, sweating, rapid heart rate, nausea, vomiting, weight loss, weakness, chest pain and anxiety.
When the tumour grows outside the adrenal gland, it is known as a paraganglioma. High levels of stress hormones can lead to life-threatening high blood pressure, heart failure, and stroke.
The FDA granted the application Fast Track, Breakthrough Therapy and Priority Review designations to speed the approval process for this aggressive type of cancer.
A clinical phase 2 trial of the medication involved 68 patients who experienced a 50% or greater reduction of all antihypertensive medications lasting for at least six months. This endpoint was supported by the secondary endpoint, overall tumour response determined via traditional imaging criteria.
Dr Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said, “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 tumour-related symptoms such as high blood pressure.
“Patients will now have an approved therapy that has been shown to decrease the need for blood pressure medication and reduce tumour size in some patients.”
Dr Daniel Pryma, associate professor of radiology & radiation oncology and the trial’s lead investigator at the University of Pennsylvania, called the drug a “true breakthrough” for patients enduring this rare cancer.