Radioactive Iobenguane I 131 Receives FDA Approval for Pheochromocytoma and Paraganglioma Treatment

On 30 July 2018, the US Food and Drug Administration FDA, for rare forms of neuroendocrine tumors including pheochromocytoma and paraganglioma treatment, approved iobenguane I 131 (brand name Azedra), a radioactive drug.

Approval was granted for disease that is locally advanced or has developed metastasis and cannot be surgically removed, in patients requiring systemic treatment who are older than 12 and have pheochromocytoma or paraganglioma. Patients must also test positive on the nuclear medicine imaging test iobenguane scintigraphy scanning as a requirement.

Administered by intravenous injection, this radioactive drug is, for this stage of pheochromocytoma and paraganglioma, the first FDA-approved drug of its kind.

Pheochromocytomais an adrenal gland tumor causing overproduction of stress hormones such as epinephrine and norepinephrine, leading to hypertension, headaches, irritability, palpitations and sweating. Paraganglioma is the same neuroendocrine tumor type but arises outside the adrenal glands in nerve clusters called paraganglia.

Details of the Study Leading to FDA Approval of Iobenguane I 131

Iobenguane I 131's effectiveness was demonstrated in 68 patients without a chance of cure through standard treatment, in a phase-2 clinical trial providing the evidence.

According to the results;

• A total of 17 patients (% 25) reduced all blood pressure medications for at least 6 months or longer by %50 or more. This was also the study's main effectiveness target (primary endpoint).
• Among 68 patients, 15 (%22) had an objective (measurable) response.
• Most patients had pheochromocytoma (% 71).

In iobenguane I 131 trials, serious effects reported in more than %10 of patients included these adverse effects lymphopenia, neutropenia, thrombocytopenia, fatigue, anemia, nausea, dizziness, hypertension and vomiting. Myelodysplastic syndrome or acute leukemia also developed in %6.8 of patients receiving a therapeutic dose of this radioactive agent (see secondary cancers).

In the study, iobenguane I-131 was initially administered at 111 to 222 MBq, followed by a therapeutic 296 MBq / kg dose thereafter. Pheochromocytoma is very rare, affecting, per 100 thousand people, 0.8. It also causes %0.2 of all hypertension. Most of these rare tumors are treated surgically, but until now no effective systemic treatment existed for inoperable patients or those whose blood pressure could not be controlled with medication. Patients now have another option proven to reduce antihypertensive medication needs and, in some cases, tumor size...

We will follow and share the approval process for iobenguane I-131 in our country.

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