The approval is for the intravenous use of the Azedra injection in patients whose adrenal gland tumors are unresectable and have spread beyond the original tumor site and need systemic anticancer therapy. The indication covers both adults and adolescents aged 12 and older.
According to the FDA, Azedra is now the first drug it has approved to treat the rare adrenal tumors.
FDA Center for Drug Evaluation and Research, Office of Hematology and Oncology Products acting director Richard Pazdur 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 tumor-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 tumor size in some patients.”
The approval of Azedra was based on the results of a pivotal phase 2 trial in 68 patients. The trial measured the number of patients who had a 50% or greater reduction of all antihypertensive medications lasting for a minimum of six months.
It met the primary endpoint, with 17 of the 68 evaluable patients experiencing a 50% or greater reduction of all antihypertensive medication for at least six months. Overall tumor response, a key secondary endpoint of the trial, was reached in 15 of the enrolled patients.
Progenics CEO Mark Baker said: “AZEDRA has been shown to decrease the need for blood pressure medication and reduce tumor size in some patients. We are extremely grateful to the patients, their families and the investigators who participated in AZEDRA’s clinical development program.”
The FDA said that as Azedra is a radioactive therapeutic agent, it comes with a warning regarding radiation exposure to patients and family members, which should be minimized while the patient is receiving the injection.