AZEDRA addresses both
essential goals of advanced
pheochromocytoma and
paraganglioma treatment

Primary Endpoint:
Antihypertensive
Medication Reduction

Secondary
Endpoint:
Tumor Control

AZEDRA demonstrated significant
and sustained reduction of
hormonal symptoms

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

25%

of patients treated with AZEDRA
achieved the primary endpoint
(n=17/68, 95% CI: 16%-37%)

32%

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
Medication Reduction†

Duration of Antihypertensive Medication Reduction with AZEDRA®. Duration of Antihypertensive Medication Reduction with AZEDRA®.
  • 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)

AZEDRA® demonstrated tumor reduction in 22% of patients. AZEDRA® demonstrated tumor reduction in 22% of patients.
  • 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

Most AZEDRA® patients had a reduction in tumor size during the 12-month evaluation. Most AZEDRA® patients had a reduction in tumor size during the 12-month evaluation.

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.

Download AZEDRA efficacy information

AZEDRA has an established safety profile

Adverse reactions occurring in ≥10% of patients who received a therapeutic dose of AZEDRA in clinical trials

Adverse reaction All
grades,a (%)		 Gradesa 3-4(%)
Hematologicb
Lymphopenia 96 78
Anemia 93 24
Thrombocytopenia 91 50
Neutropenia 84 59
Gastrointestinal
Nausea 78 16
Vomitingc 58 10
Dry mouth 48 2
Sialadenitisd 39 1
Diarrhea 25 3
Abdominal paine 23 6
Constipation 19 7
Oropharyngeal pain 14 0
Dyspepsia 10 0
General
Fatiguef 71 26
Pyrexia 14 2
Injection site pain 10 0
Hyperhidrosis 10 0
Alopecia 10 0
Infections
Upper respiratory
tract infectiong		 16 2
Urinary tract infection 11 1
Investigationsb
Increased international
normalized ratioh		 85 18
Increased blood
alkaline phosphatase		 53 5
Increased aspartate
aminotransferase		 50 2
Increased alanine
aminotransferase		 43 2
Metabolism and nutrition
Decreased appetite 30 5
Dehydration 16 4
Decreased Weight 16 1
Musculoskeletal and connective tissue disorders
Back pain 17 2
Pain in extremity 15 0
Nervous system
Dizzinessi 34 13
Headache 32 6
Dysgeusiaj 24 1
Respiratory, thoracic, and mediastinal disorders
Cough 18 0
Dyspnea 18 7
Vascular
Hypotension 24 4
Hypertensionk 20 11
Tachycardia 10 3
NCI CTCAE version 3.0. Based on laboratory data. Includes vomiting and retching. Includes sialoadenitis, salivary gland pain, and salivary gland enlargement. Includes abdominal pain, abdominal pain upper, and abdominal pain lower. Includes fatigue and asthenia.
Includes upper respiratory tract infection, sinusitis, rhinorrhea, upper airway cough syndrome, and nasopharyngitis. Only assessed in Study IB12B (N=68). Includes dizziness and dizziness postural. Includes dysgeusia, hypogeusia, and ageusia. Includes blood pressure increase and hypertension.

No secondary malignancies have been reported among patients who received AZEDRA as first-line systemic therapy as of 52 months following study completion.

Download AZEDRA safety data

Clinical practice guidelines recommend high-specific-activity (HSA) iobenguane I 131 (AZEDRA) for the treatment of advanced PPGL

National Comprehensive Cancer Network® recommendation for AZEDRA®.

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® recommendation for AZEDRA®.

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.