FDA News

FDA approves Azedra for rare adrenal gland tumors

The FDA approved iobenguane I 131 for adults and children with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require anticancer therapy.

The FDA based the approval of iobenguane I 131 (Azedra, Progenics Pharmaceuticals) — a radiotherapeutic — on an open-label, single-arm study.

The analysis included 68 patients aged 12 years or older with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma.

Seventeen patients (25%; 95% CI, 16-37) experienced a 50% or greater reduction of all antihypertensive mediation for at least 6 months. Researchers observed overall tumor response in 22% (95% CI, 14-33) of patients, with 53% achieving a response duration of at least 6 months.

The most common grade 3 or grade 4 adverse reactions included lymphopenia, neutropenia, thrombocytopenia, fatigue, anemia, increased international normalized ratio, nausea, dizziness, hypertension and vomiting.

In a pooled safety population, 6.8% of patients who received a therapeutic dose of iobenguane I 131 developed myelodysplastic syndrome or acute leukemia.

FDA granted this application priority review, orphan product, fast track status and breakthrough therapy designation.

The FDA approved iobenguane I 131 for adults and children with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require anticancer therapy.

The FDA based the approval of iobenguane I 131 (Azedra, Progenics Pharmaceuticals) — a radiotherapeutic — on an open-label, single-arm study.

The analysis included 68 patients aged 12 years or older with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma.

Seventeen patients (25%; 95% CI, 16-37) experienced a 50% or greater reduction of all antihypertensive mediation for at least 6 months. Researchers observed overall tumor response in 22% (95% CI, 14-33) of patients, with 53% achieving a response duration of at least 6 months.

The most common grade 3 or grade 4 adverse reactions included lymphopenia, neutropenia, thrombocytopenia, fatigue, anemia, increased international normalized ratio, nausea, dizziness, hypertension and vomiting.

In a pooled safety population, 6.8% of patients who received a therapeutic dose of iobenguane I 131 developed myelodysplastic syndrome or acute leukemia.

FDA granted this application priority review, orphan product, fast track status and breakthrough therapy designation.