The U.S. Food and Drug Administration (FDA) approved iobenguane I 131 injection for the treatment of patients ≥12 years with pheochromocytoma or paraganglioma—rare tumors of the adrenal gland—that are unresectable, have spread beyond the original tumor site, and require systemic therapy. This is the FDA-approved therapy available for this type of tumor.
The approval was based on the results of a single-arm, open-label trial that included 68 patients. The study met its primary endpoint: 17 patients (25%) experienced a ≥50% reduction of all antihypertensive medication for at least 6 months, and 15 patients (22%) achieved a tumor response.
— FDA Oncology (@FDAOncology) July 30, 2018
The most common severe adverse events were lymphopenia, neutropenia, thrombocytopenia, fatigue, anemia, increased international normalized ratio, nausea, dizziness, hypertension, and vomiting.
FDA approves Progenics' treatment for rare adrenal gland tumors https://t.co/epzfVzVbxx
— Reuters Health (@Reuters_Health) July 30, 2018
Iobenguane I 131 has a risk of radiation exposure to patients and family members, and this risk is greater in pediatric patients. Other warnings and precautions include risk of myelosuppression, underactive thyroid, elevated blood pressure, renal failure or kidney injury, and pneumonitis.
Myelodysplastic syndromes and acute leukemias were observed in patients who received this treatment, and researchers will continue to monitor and study this risk.