AZEDRA addresses both
essential goals of advanced
AZEDRA demonstrated significant
and sustained reduction of
The primary endpoint in the Phase II trial for AZEDRA was defined as a 50% reduction, including discontinuation, of antihypertensive medication for at least six months.
AZEDRA was proven to reduce the need for antihypertensive medication for a significant duration
of patients treated with AZEDRA
achieved the primary endpoint
(n=17/68, 95% CI: 16%-37%)
of patients treated with two
doses of AZEDRA achieved the primary endpoint (n=16/50)*
Improved primary endpoint response in subjects who received two doses could, in part, be confounded by the possibility that subjects remaining in the study may have had less severe disease at baseline and/or had tolerated the treatment better. Analysis is also limited by the small number of patients involved and limited conclusions can be drawn from the analysis of response by number of doses received.
AZEDRA provided sustained
clinical benefit for patients
Duration of Antihypertensive
- Sixteen patients were able to reduce their use of antihypertensive medication by 50%, but did not meet the primary endpoint duration of 6 months (median,
1.8 months, range 0.1-5.0 months).
- Thirty-five patients did not achieve a 50% reduction in their use of antihypertensive medication.
Among patients treated with at least one dose of AZEDRA (N=68), 33 were able to reduce their antihypertensive medication by ≥50%, and 17 of these maintained this reduction for six months or longer.
AZEDRA was proven to produce durable reductions in tumor size
The secondary endpoint in the Phase II trial for AZEDRA was overall tumor response by RECIST 1.0.
AZEDRA demonstrated tumor reduction in 22% of patients, as determined by a secondary endpoint
Best Confirmed Tumor Response (N=68)
- Patients achieved comparable responses regardless of prior treatment with I-131 MIBG or antineoplastic chemotherapy
- Partial responses increased at each of the 3-, 6-, 9-, and 12-month post-treatment evaluations
- Patients who did not meet the primary endpoint were still able to achieve tumor response
Overall tumor responses as measured by RECIST 1.0 included:
- Partial response: ≥30% decrease in the sum of longest diameters (SLD) of target lesions
- Stable disease: Neither ≥30% decrease nor ≥20% increase in SLD of target lesions observed
- Progressive disease: ≥20% increase in SLD of target lesions
- No complete responses were observed
Most AZEDRA patients experienced some reduction in tumor size from baseline during the 12-month evaluation
Maximum Confirmed Reduction in Tumor Size by RECIST 1.0 Criteria
Of the 68 patients treated, 56 are shown who had at least one post-baseline assessment and disease measurable by RECIST 1.0 criteria.
92 percent of patients with evaluable disease who were treated with AZEDRA had a partial response or stable disease as the best objective tumor response within 12 months (n=59/64).*
Due to the natural progression of PPGL, patients may experience SD in the absence of treatment, so it cannot be conclusively determined if SD was the result of treatment with AZEDRA.
Among patients who had at least one documented site of metastatic disease (n=64). Metastatic sites were not evaluated at baseline for four patients who received at least one dose of AZEDRA (N=68).
AZEDRA is the first and only FDA-approved therapy for iobenguane scan positive, unresectable, locally advanced or metastatic PPGL.
AZEDRA has an established safety profile
Adverse reactions occurring in ≥10% of patients who received a therapeutic dose of AZEDRA in clinical trials
|Metabolism and nutrition|
|Musculoskeletal and connective tissue disorders|
|Respiratory, thoracic, and mediastinal disorders|
No secondary malignancies have been reported among patients who received AZEDRA as first-line systemic therapy as of 52 months following study completion.
Clinical practice guidelines recommend high-specific-activity (HSA) iobenguane I 131 (AZEDRA) for the treatment of advanced PPGL
NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) recommend high-specific-activity (HSA) iobenguane I 131 (AZEDRA) as a category 2A treatment option for patients with prior positive MIBG scan with locally unresectable or distant metastatic PPGL (secreting tumors). HSA iobenguane I 131 is the first and only FDA-approved therapy option for iobenguane scan positive advanced PPGL recognized by the NCCN Guidelines®.
The North American Neuroendocrine Tumor Society (NANETS) Consensus Guidelines recommend HSA I-131 MIBG (AZEDRA) as a treatment option for patients with PPGL who require systemic therapy and have MIBG‑avid disease.
Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Neuroendocrine and Adrenal Tumors V.1.2022. © National Comprehensive Cancer Network, Inc. 2022. All rights reserved. Accessed June 24, 2022. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.