A new DRUG TRIALS SNAPSHOT is now available
Drug Trials Snapshots: AZEDRA
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the AZEDRA Package Insert for complete information.
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the AZEDRA Package Insert for complete information.
AZEDRA (iobenguane I 131)
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Progenics Pharmaceuticals, Inc.
Approval date: July 30, 2018
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Progenics Pharmaceuticals, Inc.
Approval date: July 30, 2018
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
AZEDRA is a radioactive drug for the treatment of patients 12 years and older who require systemic treatment (drugs that spread throughout the body to treat cancer cells) for tumors called pheochromocytomas or paragangliomas that have spread, or cannot be removed with surgery.
Pheochromocytoma is a rare tumor of the adrenal gland. When this type of tumor occurs outside the adrenal gland, it is called a paraganglioma. Both tumors increase the production of stress hormones (called epinephrine and norepinephrine), leading to high blood pressure and symptoms such as headaches, irritability, chest pain or anxiety.
How is this drug used?
AZEDRA is injected into a vein (intravenous) by a healthcare provider. The first injection is given to determine the treatment dose. After the first injection, two treatment doses are administered 90 days apart.
What are the benefits of this drug?
Out of 68 patients who received AZEDRA, 17 patients (25%) experienced at least a 50% reduction (including discontinuation) of all blood pressure medication for at least six months.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: AZEDRA worked similarly in males and females.
- Race: The majority of patients were White. The number of patients in other races was limited. Therefore, differences among race could not be determined.
- Age: The number of patients older than 65 years was limited. Therefore, differences among age groups could not be determined.
What are the possible side effects?
AZEDRA may cause serious side effects including radiation exposure, decreased or abnormal bone marrow production of blood cells, increased risk of cancer, underactive thyroid, elevations in blood pressure, damage to the kidneys, lung inflammation, risk to a fetus, and infertility.
The most common side effects were low blood cells counts, and fatigue.
Were there any differences in side effects among sex, race and age?
- Sex: The occurrence of side effects was similar in males and females.
- Race: The majority of patients were White. The number of patients in other races was limited; therefore, differences in the occurrence of side effects among races could not be determined.
- Age: The number of patients older than 65 years was limited. Therefore, differences in side effects among age groups could not be determined.
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved AZEDRA based on evidence from two clinical trials (Trial 1 /NCT01413503 and Trial 2 /NCT00874614) of 88 patients with pheochromocytomas or paragangliomas that have spread and cannot be removed with surgery. The trials were conducted at 13 sites in the United States.
Both trials were used to evaluate safety of AZEDRA. The safety population is presented below. Only Trial 1 was used to evaluate the benefits of AZEDRA. Demographics of these patients are presented in Table 7, under the MORE INFO section.
Figure 1 summarizes the percentage of patients by sex in the combined clinical trials used to evaluate safety.
Figure 1. Baseline Demographics by Sex (safety population)
FDA Review
Figure 2 summarizes the percentage of patients by race in the combined clinical trials used to evaluate safety.
Figure 2. Baseline Demographics by Race (safety population)
FDA Review
Table 1. Demographics of Clinical Trials by Race
| Race | Number of Patients | Percentage of Patients |
|---|---|---|
| White | 66 | 75% |
| Black or African American | 17 | 19% |
| Asian | 5 | 6% |
FDA Review
Figure 3 summarizes the percentage of patients by age in the clinical trials used to evaluate safety.
Figure 3. Baseline Demographics by Age (safety population)
FDA Review
How were the trials designed?
The benefit and side effects of AZEDRA were evaluated in two clinical trials that enrolled patients with specific tumors called pheochromocytomas or paragangliomas that have spread and cannot be removed with surgery. The presence of all tumors was confirmed with an imaging scan.
Trial 1 enrolled adult patients who were 18 years and older. All patients received a starting dose of AZEDRA. Afterwards, patients were assigned to one of four strengths of AZEDRA that was given as a single injection within 7 – 28 days after the starting dose. Patients were evaluated for side effects.
Trial 2 enrolled patients who were 12 years and older. Patients received a starting dose of AZEDRA followed by 2 treatment doses 3 months apart. Patients were evaluated for side effects and benefit. The benefit of AZEDRA was assessed by the proportion of patients who achieved at least a 50% decrease in the use of blood pressure medications for 6 months.
GLOSSARY
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.
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